TIF Magazine - issue 60

Thalassaemia International Federation

TIF MAGAZINE

ISSUE No 60 December 2011

Published by the Thalassaemia International Federation | www.thalassaemia.org.cy

Cyp r u Lim s as

sol

KNOWLEDGE IS OUR POWER

UNITY IS OUR STRENGTH

“...Thalassaemia is no longer a fatal disease,

TIF MAGAZINE December 2011

PRESIDENT’S Address

it is a chronic disease—one that is both preventable and treatable, with high rates of survival where there is free access to quality care.

This is a truly exceptional development.”

Dear friends, The start of a new year offers a chance to reflect on recent achievements, while gathering ourselves for the challenges that lie ahead.

After two and a half decades of painstaking work, TIF activities have entered a new phase. Having established itself as a leading patient-driven, activist organisation, TIF is now in a position to push for major improvements in the lives of patients. But that is not enough. TIF must also ensure that our efforts have a measurable impact, on policy-makers and on patients, their families and the medical staff that care for them.

Guidelines for the treatment and prevention of thalassaemia are now available from every national association, and sustained attempts to increase the number of reference centres around the world are ongoing.

However, a major challenge is to give greater voice to patients by educating them about their disease, facilitating their ability to better manage the demands of living with thalassaemia—to be involved in the decisions that affect them and to lobby for their rights to services, to education, to employment. The guiding principle is, as the slogan has it: "Nothing for us, without us."

In many ways, TIF has been ahead of the curve here.

Patient-centred healthcare, for example—a concept that has recently assumed centre stage in Europe—is a longstanding TIF demand, as are the issues of patient safety and patient rights. The 2nd pan-European Conference, held in Berlin in March 2010, had as its motto "Knowledge is Power". TIF has always said the same.

TIF's Expert Patients Programme aims to further educate patients about thalassaemia and broader public health issues, empowering them to act as valuable part-

ners in policy-making. A core group of five expert patients met in London in November 2011, and the programme will soon expand to include a wider group spread around the world.

TIF is also realising the fruits of efforts to leverage its access to the European Union, taking a lead role in a number of international projects aimed at improving the management of chronic conditions. By establishing thalassaemia in the broader class of haemoglobinopathies—diseases of the blood—TIF has ensured that thalassaemia joins a widely-discussed category of diseases that affect millions of people. The need now is to integrate the management of thalassaemia in still more national healthcare systems.

The big story, though, is about thalassaemia itself. As a result of TIF's work with national thalassaemia associations, scientists, patients and their families, the natural history of thalassaemia has changed forever.

Thalassaemia is no longer a fatal disease, it is a chronic disease—one that is both preventable and treatable, with high rates of survival where there is free access to quality care. This is a truly exceptional development.

The second major challenge, then, is to persuade policymakers everywhere that the most cost-effective way of dealing with thalassaemia and other haemoglobinopathies is to recognise such diseases as significant public health issues, and treat them accordingly.

TIF is proud of the great strides made by all our member associations in 2011, encouraged by the fighting spirit of thalassaemia patients and their families. Their remarkable energy and determination is an inspiration to us all.

Best wishes for a happy, healthy 2012.

Panos Englezos TIF President

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TIF MAGAZINE December 2011

CHIEF EDITOR’S Address

In 2012 two major regional conferences will be the driving force of TIF’s activities in the Asian and European regions.

As we enter 2012, it is time to reflect on the past year and plan ahead for the future.

moting national prevention programs and most importantly, strengthening research.

The last months have been extremely busy, exciting and productive, with numerous activities taking place all over the world. Indeed we have undertaken, participated in and supported meetings and conferences in many parts of the world including Algeria, Morocco, Philippines, Myanmar, Jordan, Bulgaria, Albania, and the Russian Federation.

An overview of all the significant events of the previous year can be found in this issue but also a sneak preview of the exciting events to come. In 2012 two major regional conferences will be the driving force of TIF’s activities in the Asian and European regions.

We have made significant progress in consolidating our collaborative relationships with important organisations in Europe and across the world. Particularly with patient/parent associations that we had the opportunity to see during the International Conferences on Thalassaemia and Other Haemoglobinopathies that took place last May in Antalya, Turkey. Τhe Antalya International Conferences were undoubtedly the highlight of the year for the global thalassaemia community. They provided, as always, a delight to witness in person the gathering of such numbers of patients, parents and dedicated medical professionals.

This zest was also evident in the celebrations of World Thalassaemia Day that took place on the 8th of May. The theme this year was ‘Equal Chance to Life’ and was selected to highlight the health inequalities experienced by hundreds of thousands of patients across the world.

This issue of TIF Magazine contains a selection of reports and activities that took place internationally, embracing this theme: fighting against the violation of patient rights.

2011 marked also the establishment of a new prestigious and highly significant international award – the Sultan Bin Khalifa International Award. This important award was born by the generosity of His Highness Sheikh Dr. Sultan Bin Khalifa Al Nahyan, son of the President of the United Arab Emirates. It is a great honour and privilege for TIF and all of us who work for the cause of thalassaemia to have the recognition of such eminent persons. The global award constitutes a milestone in the promotion of TIF’s mission in raising awareness and pro-

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The postponement of the 1st Pan-Asian Conference on Haemoglobinopathies in November has meant that this will take place now on 8 – 10 February 2012 in Bangkok, Thailand. Those who will attend what is expected to be an impact-making meeting will benefit from its targeted programme which with the support and contribution of key policy markers and medical specialists aims to map the situation and progress of more than 15 South – East Asian and Western Pacific countries.

Furthermore, TIF has already set wheels in motion for the organisation of the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias. This regional conference will take place on 24 – 26 October 2012 in Limassol, Cyprus thus taking advantage not only of the wonderful sunny weather but also of the geographical crossroads at which Cyprus rests upon – joining the east with the west. This is an event that I am sure we all await with excitement, will be the undisputed high point of the year. This grand educational event has been placed under the auspices of the Cyprus Government during its European Presidency and will feature a wealth of topics both for medical professionals as well as for patients, policy-makers and the wider health community.

TIF’s activities in 2012 will focus on further strengthening our collaboration with member associations and medical networks and deepening our impact at the regional level, building on a wide-ranging portfolio of projects developed over recent years.

On behalf of the TIF office, I wish all our readers a healthy and productive 2012.

Dr Androulla Eleftheriou Executive Director

Chief Editor Androulla Eleftheriou, PhD

TIF MAGAZINE December 2011

EDITORIAL Team

Editor Helen Perry Contributors Dr Saeed Jaafar Al Awadhi Dr Ali Taher Dr Khaled Musallam Dr Chi Kong Li Dr Mehran Karim Dr Michael Angastiniotis Mrs Phoebe Katsouris Ms Lily Cannon

Board of Directors Panos Englezos President Shobha Tuli Vice President Loizos Pericleous Secretary Riyad Elbard Treasurer Her Highness Sheikha Sheikha Bint Seif Al-Nahyan TIF MAGAZINE Thalassaemia International Federation

George Constantinou

PO Box 28807, 2083 Nicosia, Cyprus Tel: +357 22319129 / 22319134 Fax: +357 22314552 E-mail: thalassaemia@cytanet.com.cy www.thalassaemia.org.cy

Anton Skafi

Designed by Chromasyn Printed in Nicosia

Fatemeh Hashemi

Reproduction of material published in TIF Magazine for educational purposes is encouraged, provided it is accompanied by the following attribution “... according to TIF Magazine, the official newsletter of the Thalassaemia International Federation�.

The contents of any scientific article or presentation of any material by manufacturers does not imply the expression of any specific companies or products does not imply that they are endorsed or recommended by TIF in preference to others. The contents express the opinions of the authors who alone are responsible for the views expressed. TIF does not accept any legal responsibility for their contents.

Loris Angelo Brunetta Michael Michael Robert (Bob) Ficarra Saeed Jafaar Al-Awadhi Mouna Haraoui Ivan Dimitrov Ivanov Duru Malyali Ramli Mohd Yunus Christina Stephanidou Nailya Guliyeva

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TIF MAGAZINE December 2011

TABLE of Contents 6

Board Members Corner

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TIF Projects

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TIF Activities

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TIF International Conferences 12th International Conference on Thalassaemia and the Haemoglobinopathies and 14th TIF International Conference for Patients and Parents / Antalya, Turkey

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TIF Delegation Visits

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News from our Members Around the World

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TIF Regional Conferences 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias 24-26 October 2012 / Limassol, Cyprus

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World Thalassaemia Day

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Featured Articles FOCUS: Thalassaemia & Employment

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Medical Focus Synthetic blood: An end to blood drives?

A closer look at β-thalassaemia intermedia How to select iron chelators in 2012 Hydroxyurea management in β-thalassaemia intermedia

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TIF Regional Conferences 1st Pan-Asian Conference on Haemoglobinopathies, Bangkok, Thailand (8-10 February 2012)

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Upcoming Events

TIF MAGAZINE December 2011

BOARD MEMBERS’ Corner Her Highness Sheikha Sheikha Bint Saif Al Nahyan Her Highness Sheikha Sheikha Bint Saif Al Nahyan has been a driving force in raising awareness and promoting excellence in the treatment of haemoglobinopathies in the United Arab Emirates (UAE).

With a particular interest in the well-being of children, Her Highness is also Chairman of Make-AWish UAE, and a leading supporter of other local and international charities.

Prestigious new international award for thalassaemia A major new award for significant contributions to thalassaemia was announced in April 2011, backed by His Highness Sheikh Sultan Bin Khalifa Al Nahyan, son of the President of the United Arab Emirates (UAE), with the support of the Thalassaemia International Federation (TIF).

The Sheikh Sultan International Thalassaemia Award, worth around $1 million, is sponsored by His Highness Sheikh Al Nahyan, with TIF acting as expert adviser. TIF President Panos Englezos will serve as Vice President of the Award’s administrative Board of Trustees.

The prize aims to raise awareness about thalassaemia and other haemoglobin disorders by celebrating

outstanding achievements in the field, and will be awarded every two years. Winning individuals and/or organisations will be announced during the biennial TIF international thalassaemia conferences, followed by an award ceremony in Abu Dhabi.

The UAE is proud to take the lead in establishing such a prestigious prize, following its own significant advances in the management of thalassaemia and in the broader medical arena. It is hoped that this award will play a major part in strengthening efforts to educate the public about thalassaemia, as well as providing further motivation and encouragement to all those working to improve the lives of thalassaemia patients.

Saeed Jaafar Al Awadhi has been a Board Member of the Emirates Thalassemia Society since 2003, where he also serves as Financial Manager, in addition to his role as an officer of the Dubai Health Authority Thalassaemia Centre. Saeed is a member of the Sultan Bin Khalifa International Thalassaemia Award Executive Committee, and attended the recent 12th International Conference on Thalassaemia & Haemoglobinopathies and the 14th TIF International Conference for Patients and Parents. Saeed has been elected to the TIF Board of Directors for a term of four years.

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TIF MAGAZINE December 2011

TIF Projects TIF is involved in a number of major projects across a range of areas relating to thalassaemia, from ensuring good blood donor management to promoting the further education of specialists in the haemoglobinopathies and developing systems for the electronic exchange of patient data.

Donor Management in Europe—DOMAINE DOMAINE is a European Commission-funded project to identify and promote good blood donor management in practice. TIF was the only patient-led organisation selected to take part in this important research.

The DOMAINE project aimed to devise ways of ensuring the safe and sufficient supply of blood, by comparing national experiences across Europe to establish good donor management practice. The research concentrated on donor recruitment and retention strategies, and blood bank policy regarding patients requiring long-term transfusion.

The first phase of the project involved a survey of donor management practice in a number of countries. In the

Following a preliminary training workshop in Slovenia in April 2011, the DOMAINE training committee organised a two-day, interactive training workshop in Lisbon, Portugal, on 17-19 June 2011. The workshop involved 25 participants from across Europe, all of whom had advised on the compilation of the manual. Participants were then asked to complete a questionnaire evaluating the material. The results of the questionnaire are currently being analysed and are expected to be made public soon.

Further details regarding the DOMAINE project can be found at www.domaine-europe.eu.

MSc in Haemoglobinopathy faces challenges

Epidemiology project approaches completion

TIF was instrumental in the establishment of an MSc in Haemoglobinopathy at University College London (UCL), which has been recognised by the authorities in Cyprus as a long-distance learning Masters degree. TIF is now working to obtain similar recognition for the course from the Ministries of Health of a number of Middle Eastern countries, including Syria, Oman, Qatar, Kuwait, Saudi Arabia and the UAE.

TIF’s epidemiology project is coming to a close after months of data-gathering. A final analysis of the information collected was presented to the TIF Board of Directors in November 2011, covering more than 80 countries affected by β-thalassaemia and HbE. Data included details of carrier rates and the number of anticipated births each year, as well as information regarding national health systems and correlations between GDP, per capita health expenditure and disease prevalence, highlighting focal points in the global effort to manage haemoglobin disorders.

Ranked as one of the world's top four universities, UCL is an outstanding institution of further education. The MSc has been extremely successful and the course directors have been awarded the Provost's Teaching Award for excellence in teaching. It is therefore very important that countries with a high rate of thalassaemia and sickle cell disease recognise this leading programme as a crucial tool in the further education of their health care professionals.

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second phase a manual was compiled, setting out guidelines for good donor management, includes tools and examples of incentives to motivate future and current donors to become regular donors.

The final phase of the project entailed training professionals in the use of the manual.

The second phase of the project will get underway in 2012, completing the collection of data on sickle cell disease and HbH. The data compiled will play a critical role in shaping future TIF work, as well as offering essential information to leading United Nations agencies such as the WHO, in their efforts to establish these disorders on national health agendas.

Thalassaemia Specific Electronics Communications The effective follow up of disease progression and documentation of the impact of treatment in thalassaemia as for every other chronic disease has always depended on accurate clinical records.

As technology moves more and more into health, the value of the use of electronic formats has been widely recognised. Electronic records are ideally suited to the long-term collection and evaluation of such data. In an effort to support the Cyprus Thalassaemia Centre's efforts to transfer paperbased records to an electronic format, TIF launched the Thalassaemia Specific Electronics Communications project—a pilot study using a specially designed software to upload more than 40 years’ patients data. The results of the study were presented at a workshop held in Cyprus on 20 October 2011 and raised considerable interest and confirmed the immense value of this project.

TIF is now working on expanding this project across Cyprus, with the eventual aim of sharing this software with other haemoglobinopathy centres around the world, facilitating data transfer and storage, as well as boosting opportunities to network.

New TIF Publications TIF is two publish two new books in 2012, as well as revised editions of textbooks on the prevention of haemoglobinopathies, updated to include advances in laboratory techniques.

TIF MAGAZINE December 2011

TIF Projects

The first of the new books is Thalassaemia Guidelines for the Accident and Emergency Department—a manual covering the management of thalassaemia in emergency room situations, compiled by world experts in the fields of thalassaemia and emergency medicine. The manual offers medical staff a convenient, easy-to-use guide to diagnosis and practical action in the event of a thalassaemia patient requiring emergency care. The handbook lists common presenting symptoms, such as dyspnoea, chest pain, syncope and oedema, using flow charts and concise text to convey essential points. It will be made available to emergency hospital units and primary care clinics.

The second book, Guidelines for Nurses in a Haemoglobinopathy Centre, is a textbook for nurses working in specialised haemoglobinopathy centres compiled by a group of nurses working in Lebanon, Cyprus and the UK. The book reflects recognition of the special role of nurses in centres dealing with thalassaemia and sickle cell disease, where they offer a combination of expert knowledge and long-term patient support.

As such, the book aims not only to improve nursing care but also to attract nurses to work in such centres over long periods, to ensure continuity of care.

ENERCA- the European Network for Rare and Congenital Anaemias TIF has also played a leading role as a partner in the very important European Commission-funded project ENERCA (European Network for Rare and Congenital Anaemias).

The project aims to bring experts together to improve public health services in the field of rare anaemias and more particularly, to identify criteria and support the national and European efforts towards establishing or strengthening existing Centres of Expertise in rare anaemias, including haemoglobinopaties across the EU. TIF has been a participant collaborator in this project for the last three years. Thalassaemia is a rare anaemia in most of in Europe and ENERCA is a project which aims to create reference networks in Europe to share expertise, disseminate knowledge for both professionals and patients and to maintain epidemiological surveillance. The project is ongoing but already has produced interesting

results such as a report on the legal framework and ramifications for the establishment of expert centres, including matters of confidentiality of patients’ data and samples. It has produced and published recommendations on the clinical management of sickle cell disease, while recommendations for thalassaemia are being prepared.

Also completed are recommendations for the prevention of haemoglobin disorders which are awaiting publication.

For more information about this and related activities, visit the project website: www.enerca.org. In collaboration with the Bulgarian Thalassaemia Organisation, ENERCA has successfully organised a workshop on rare anaemias in Sofia on the 19th - 20th of November 2011.

The final result of this phase of the project will be a white paper on the requirements of an expert or reference centre for rare anaemias. In formulating the recommendations the expectations of patients of such a centre will be considered according to patient responses to a questionnaire prepared

by TIF. This report is expected to be ready in 2012.

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TIF MAGAZINE December 2011

TIF Projects Chain of Trust ‘Chain of Trust’ is the code name of a European funded project, developed by the European Patients Forum (EPF) in which TIF participates. The project focuses on building confidence and acceptance of telehealth solutions among patients and health professionals. Involved in the project are professional and patient organisations, The programme has investigated attitudes and practices but has also conducted workshops in several countries including Norway, Holland, Latvia and soon in Greece, to sensitise and inform the various groups on the importance of telehealth. The project is expected to be completed by the end of 2011.

Expert Patients Programme

A

This project is one of the most important projects TIF has ever undertaken, aiming to empower patients to take meaningful and timely decisions about issues affecting their healthcare and quality of life. The main component of the project has been the preparation of educational material, which has been reviewed by an international expert patient group as well as by a panel of scientists. The final draft is expected to be presented 17:47 Page at the first official meeting of the International Core Group of Experts in London on 25 November 2011,

where the second phase of the project will be completed.

The vision of TIF is to be able to extend in the coming years widely across the world accurate and up-to-date information and knowledge to its global patient family.

Endocrinology Network The Endocrinology Network, which was established during the international conference in Antalya in May 2011, held a second meeting in Doha, Qatar on 2-3 October 2011, during a workshop on the endocrinological complications of thalassaemia. The objectives of the group include the promotion of endocrinological followup of multi-transfused patients, the education and training of more endocrinologists in this specialised field, and the promotion of collaborative research. One project discussed in Qatar is the development of guidelines for the management of endocrine complications in thalassaemia. A third meeting of the group took place in Catanzaro, Italy on 11-13 November 2011. During this meeting, a final plan of action for the group, known as ICET (International Complicanze Talassemia), was agreed and a proposal presented to TIF. The group consists mainly of endocrinologists, as well as thalassaemia 1 specialists from Italy, Egypt, Greece, Cyprus, Qatar, Iran, India, Turkey and the USA.

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TIF MAGAZINE December 2011

TIF Activities Understaffing risks WHO work on Non-Communicable Diseases

21st Regional Congress of the International Society of Blood Transfusion

The European Genetic Alliance Network (EGAN) has joined TIF in submitting a statement to the WHO, outlining concerns over understaffing of the Human Genetics Programme.

It is the belief of both organisations that the delay in appointing a replacement for the Programme's previous coordinator, Dr Victor Boulyjenkov, risks undermining WHO work in the field of chronic genetic diseases such as the haemoglobinopathies and other blood disorders.

The joint statement of concern, addressed to the Director General of the WHO, also requested that the haemoglobinopathies receive prominence on the WHO list of NonCommunicable Diseases (NCD). A high-level meeting of the UN General Assembly on NCD was convened in September 2011.

TIF took part in the 21st Regional Congress of the International Society of Blood Transfusion (ISBT), held in Lisbon, Portugal, on 18-22 June 2011.

A key area of interest was the ISBT Working Party on Global Blood Safety, formed after the International Congress of the ISBT held in Berlin in July 2010. The objective of this group is to foster improvements in blood safety around the world, through the networking of individuals and organisations with expertise in transfusion medicine. Blood safety constitutes a major concern for multiply-transfused patients, particularly as transmission of pathogens through blood is still occurring in many regions of the world with high prevalence of thalassaemia.

Cyprus Alliance of Rare Disorders The Cyprus Alliance of Rare Disorders (CARD) took part in a workshop organised by the Steering Committee for Rare Diseases of the Cyprus Ministry of Health, held on 23-24 June 2011. TIF is a founding member of CARD, which was represented at the meeting by TIF Executive Director Dr Androulla Eleftheriou and CARD Coordinator Lily Cannon.

The workshop aimed to map the current situation regarding rare diseases in Cyprus, and included roundtable discussions on the research, prevention, treatment and diagnosis of rare diseases. A discussion on the support services offered to patients with rare diseases was led by TIF, covering rehabilitation, palliative and respite care.

The workshop was the first of a series of stakeholder events that will contribute to the drafting of a strategic national plan for rare diseases, in accordance with European Council Recommendation 2009/C 151/02 and EUROPLAN guidelines.

Addressing participants, Cyprus Minister of Health Dr Christos Patsalides stated that the creation of such a plan was essential, enabling the state to meet its obligation to support patients by improving and expanding services as necessary.

The workshop concluded that a significant amount of work remains to be done, particularly in the areas of prevention, diagnosis and treatment. The working groups are expected to meet again before the end of the year, for further deliberations.

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Joint TIF/EPHA/ASPHER Conference

A conference aimed at preparing healthcare professionals in Cyprus for the island's Presidency of the European Council, which begins in the second half of 2012, was organised by TIF, the European Public Health Alliance (EPHA) and the Association of Schools of Public Health of the European Region (ASPHER), held in Nicosia, Cyprus on 3 September 2011.

Entitled Preparing the Health Community for the Cypriot Presidency of the EU, the conference aimed to empower public health actors to engage in policy-making before and during the six months that Cyprus will hold the Presidency.

Topics included the need for EU consensus over the role of public health, and the public health capacity needed to meet identified targets. Participants included representatives of the governmental and non-governmental sectors—patients, policy makers and public and private healthcare managers.

TIF Executive Director Dr Androulla Eleftheriou opened proceedings with an overview of ASPHER’s public health competencies programme in relation to the Cyprus Presidency, while ASPHER Board Member and EPHA Vice President Chris Birt provided a particularly inspiring address on the drive needed to take part in the policymaking process.

EHA pushes innovation in haematological research TIF’s Executive Director was invited to represent patients with non-malignant haematological diseases at the 16th EHA Congress, held in London on 9 June 2011. This marked a step forward for the European Haematology Association (EHA) in bridging the gap between haematologists and patients, as the Association attempts to establish a Patient Advisory Committee. The Committee hopes to bring together patient organisations which represent patients with both malignant and non-malignant haematological diseases throughout Europe.

In continuation of this first meeting in London of patient organisations, a follow-up meeting was organised by EHA in collaboration with the European Coalition of Cancer Patients, in Brussels, Belgium on 30 -31 August 2011. TIF’s Executive Director attended this stakeholders meeting entitled ‘Haematology and the Next Decade’ and participated in the meeting with a presentation about the success story of thalassaemia in the Mediterrnean region, focusing specifically on the story of Cyprus. The meeting specifically focused on discussing the need to strengthen research funding in haematology, the revision of the clinical trials directive and the recognition of professional qualifications as a means of providing quality healthcare across Europe.

Opening the conference, Member of the European Parliament (MEP) Pawel Kowal joined fellow MEP Elzibieta Lukacijewska to highlight public ignorance of haematology as a key issue of concern. EHA continues to focus considerable effort on ensuring that haematological diseases are at the forefront of areas of interest to the European Union's research and development programme.

TIF joined by the other haematological patient organisations present at the meeting are preparing a joint position paper on the outcome of the discussion, which is to be presented to the EU Commissioner for Research and Innovation Sciences, Mairie Geoghegan-Quinn and EU Commissioner for Health and Consumers, John Dalli, urging them to take action.

Rare Diseases and the Russian Federation

TIF MAGAZINE December 2011

TIF Activities

The 2nd All-Russian Conference for Rare Diseases and Rarely Used Medical Technologies took place in St Petersburg, Russia on 21-22 April 2011. TIF was represented by two experts on the haemoglobinopathies, Dr John Porter and Dr Mary Petrou. The conference was organised under the auspices of the EUROPLAN project, which supports national health authorities to develop and implement strategies for the management of rare diseases.

The conference was followed by WHO's First Global Ministerial Conference on Healthy Lifestyles and NonCommunicable Disease Control, held in Moscow on 28-29 April 2011. The aim of the conference was to support member states in the development of programmes on healthy living and disease prevention.

A report on the resolutions of the All-Russian Conference for Rare Diseases was prepared by Svetlana Karimova, President of the National Association of Organisations of Patients with Rare Diseases. The report concluded that a commitment to establish national plans for rare diseases has gained momentum across Europe, reinforcing work by the Russian Ministry of Health to introduce a draft federal law. Recommendations in her report include the harmonisation of Russian terminology with that of the EU, and the establishment of an Expert Council for Rare Diseases and Orphan Techniques at the Russian Ministry of Health.

For its part, TIF is maintaining its efforts to make sure that haemoglobinopathies, and in particular thalassaemia, are included in the formulation of Russia's national plan for rare diseases and in the Federation's broader public health programme. We are confident that with further cooperation, the country will soon establish a register of thalassaemia patients along with screening and treatment programmes, as well as prevention strategies.

Eastern Mediterranean Region (EMRO) Guidelines for Haemoglobin Disorders

On the 27-30 June 2011, TIF participated in a meeting organised by the Regional WHO-EMRO and the Blood Disorders unit of the CDC of Atlanta, USA, held in Amman, Jordan. The meeting focused on Haemoglobinopathies and Genetic Diseases, bringing together experts and public health representatives from across the region. TIF was represented by Executive Director Dr Androulla Eleftheriou, who was invited to cover the topic of haemoglobinopathies in developing countries. The purpose of the meeting was to initiate a project for the formulation of guidelines for haemoglobin disorders in the Eastern Mediterranean, a process in which TIF will play a significant role.

During the visit, TIF also gathered epidemiological and other data, as part of its work to assess national health services for patients with thalassaemia.

(For news on TIF's Delegation Visit to Jordan, see inside.)

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TIF MAGAZINE December 2011

TIF INTERNATIONAL Conferences

12th International Conference on Thalassaemia and the Haemoglobinopathies 14th TIF International Conference for Patients and Parents Antalya, Turkey / 11-14 May 2011 This year's twin conferences on thalassaemia and the haemoglobinopathies, held in Antalya, Turkey, were the most successful yet, attracting more than 1,300 participants from over 50 countries across five continents. The four-day event, held at the WOW Kremlin Palace Hotel, was co-organised by TIF and the Thalassaemia Federation of Turkey, with the support of the Turkish Ministry of Health.

A notable feature of this year's conferences was the evenly matched numbers of patients and medical specialists, helping to encourage particularly lively discussion between the two. Another was the awe-inspiring beauty of Antalya and the exceptional hospitality of the conferences' Turkish hosts.

Abstracts of the scientific programme can be found at http://pagepressjournals.org/index.php/thal/article/view/tr.2011.s1

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Panos Englezos and George Englezos Awards The Antalya event served as the ideal forum for the award of two prestigious prizes—the Panos Englezos Award for exceptional role models in the fight against thalassaemia, and the George Englezos Award for outstanding scientific contributions to the field.

The 2011 Panos Englezos Award was presented to two outstanding individuals, Costas Kontourou and Anastasios Leventis.

The posthumous recognition of Costas Kontourou highlighted the exemplary courage and energy of a man who refused to give up the fight against thalassaemia. As a patient, Costas was a committed and tireless supporter of TIF's work, never missing an international conference or regional workshop. But perhaps more importantly, Costas had an infinite capacity for compassion and friendship, sharing a joy and zest for life with everyone he met.

Costas's much-deserved award was accepted by his beloved sister and mother, who travelled to Antalya for this very special occasion. He is much missed by the many people touched by his inspirational life.

TIF MAGAZINE December 2011

TIF INTERNATIONAL Conferences

As Chairman of the Leventis Foundation, Anastasios Leventis has shown great generosity in his support for the struggle against thalassaemia, including the establishment of an e-MSc in Haemoglobinopathies at University College London, launched in 2009. TIF and the wider thalassaemia community are greatly indebted to Mr Leventis for his unstinting encouragement of efforts to improve the knowledge and treatment of patients suffering from haemoglobinopathies. TIF is honoured to have had the opportunity to mark its appreciation of such an admirable and accomplished individual.

The 2011 George Englezos Award for an outstanding scientific contribution to the field of thalassaemia was awarded to Dr Hans Peter Schnebli, in recognition of his groundbreaking work on iron chelation. Dr Schnebli's research resulted in the development of the oral chelator Deferasirox, an FDA/EMA approved drug that has transformed the lives of so many patients around the world.

But this award recognised not just Dr Schnebli's scientific prowess but his humanity. Dr Schnebli's sensitivity to the pain and suffering of thalassaemia patients enabled him to understand just how much an oral chelator would improve their quality of life. It is thus as much for his personal qualities that TIF is proud to have presented Dr Schnebli with the 2011 George Englezos Award.

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R2-MRI The only standardised, quality-controlled, MRI-based measurement of liver iron concentration (LIC) with international regulatory clearances and approvals.

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The FerriScan® report is available within two working days, providing an LIC result, not an indirect measurement. This, together with a report of the patient’s LIC history enables clinicians to easily and accurately assess their progress.

A measurement of cardiac T2* can also be provided with a FerriScan® at selected facilities internationally, providing a comprehensive picture of body iron loading.

Please visit www.ferriscan.com or email info@ferriscan.com for further information.

TIF Delegation Visits Jordan 27-28 June 2011 TIF's participation in a WHO-EMRO meeting in Jordan in June 2011 offered an excellent opportunity for TIF Executive Director Dr Androulla Eleftheriou and a member of staff to visit thalassaemia centres and blood banks in Amman and Irbid. TIF also enjoyed a very productive meeting with members of the Jordanian Thalassaemia and Haemophilia Association—a longstanding member of TIF, and its president, Dr Basem Kiswani.

The TIF delegates, accompanied by Dr Kiswani, also met the General Secretary of Jordan's Ministry of Health, Dr Daifallah Al Louzi, with whom they discussed the current status of control strategies and management services for thalassaemia, and considered ways of strengthening TIF’s cooperation and support for further improvements. The meeting was extremely productive and TIF was very pleased to have seen evidence of significant improvements.

conference to be held in November 2011—a valuable opportunity to further strengthen TIF's relationship with the Ministry of Health, as well as to meet patients and extend its network of health professionals in the country.

TIF MAGAZINE December 2011

TIF Delegation Visits

Philippines 2-4 September 2011

Morocco 4-5 May 2011

During the course of a Delegation visit to Morocco on 4-5 May 2011, Minister of Health Yasmina Baddou and TIF Executive Director Dr Androulla Eleftheriou signed a three-year joint plan of action, aimed at establishing a national thalassaemia control programme. The move marks a major step forward in recognising the importance of haemoglobin disorders, both in terms of Morocco's national health agenda and that of the national haematology association.

The plan covers the 2011-2013 period, during which a number of services to improve the lives of thalassaemia patients are expected to be designed and implemented.

TIF was also invited to participate in a haematological

TIF's delegation visit to the Philippines in September 2011 marked a number of important firsts. This was the first such visit to the country by TIF, and was organised to coincide with the 1st National Thalassaemia Workshop for Patients and Parents, and the 1st Health Professional Workshop on Thalassaemia and Haemoglobinopathies.

The Philippines is highly affected by β-thalassaemia HbE and HbH. Aside from offering an invaluable opportunity to meet patients, parents and health professionals, the visit also represented a chance to discuss and assess the management, prevention and treatment of thalassaemia on the ground, facilitating TIF's efforts to tailor its support as needed. An important start has already been made, with the launch of a national patients’ register to establish patient numbers and carrier rates.

The visit kicked off with a meeting of representatives from the national health authority, blood banks and patient groups. TIF representative Dr Michael Angastiniotis joined Dr Anthony Calibo, Supervising Health Programme Officer at the Family Health Office of the National Centre for Disease Prevention and Control, and members of the Thalassaemia Working Group and the Philippine Society of Haematology and Blood Transfusion for broad-ranging discussions on key issues. The following day, the delegation met the Head of the National Blood Centre, Dr Bonifacio, and the Director of the Red Cross National Blood Service, Dr Christie Monina Nalupta.

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TIF Delegation Visits Day three saw the start of the 1st National Thalassaemia Workshop for Patients and Parents, bringing together patients and parents from across the country to share their concerns and network, with the final day dedicated to the 1st Health Professional Workshop on Thalassaemia and Haemoglobinopathies.

Egypt 26 September 2011 A Delegation visit to Egypt on 26 September 2011 focused on supporting efforts by the country's medical community to establish a national control programme. TIF has enjoyed a long history of cooperating with thalassaemia groups in Egypt, and welcomed the chance to visit again following a period of political instability.

While Egypt has seen many significant advances in the management of thalassaemia over recent years, carrier rates remain high and the lack of a coherent prevention strategy means that the already large number of thalassaemia patients is on the increase. The visit therefore offered a valuable opportunity to assess overall progress in the fight against thalassaemia in Egypt, with a view to further strengthening TIF's support.

Israel 5-6 October 2011 Despite an exemplary health system offering patients free and full access to the highest standards of care, there are indications of weakening compliance amongst adult thalassaemia patients in Israel. TIF's delegation visit to the country on 5-6 October 2011 therefore aimed to focus on

this issue, with a view to exploring aspects relating to psychosocial needs and social integration, including opportunities for employment and higher education.

The TIF Delegation had the opportunity to meet with internationally-renowned medical experts such as Professor Rachmilevitch – a long standing collaborator of TIF. The Delegation also visited the EMEK Medical Centre in the city of Afula medical staff and had discussions with representatives of the Galilee Society for Thalassaemia and Sickle Cell Anaemia.

The following day, the Delegation visited the Wolfson Medical Centre, the Rabin (Beilinson) Medical Centre and the Schneider Children’s Medical Centre of Israel.

Meetings were also held with representatives of the Israeli Association for Thalassaemia and Sickle Cell Disease.

Discussion centred on a number of issues, including current research on thalassaemia in Israel, the extension of MRI T2* to more countries for the monitoring of cardiac iron overload, and ways to move forward successfully transferring from paediatrics to adult haematology medicine.

The visit offered TIF the opportunity to further strengthen its commitment to supporting the thalassaemia community in Israel in a number of ways, including the development of cooperative networks of medical specialists and national thalassaemia associations, the provision of educational material and the organisation of a national thalassaemia workshop in the near future.

Send us your news

If you have important news you want to share with the world thalassaemia community, let us know! thalassaemia@cytanet.com.cy

4th Pan-American Thalassaemia Conference

By Dr Vasili Berdoukas, USA and Dr Aderson Araujo, Brazil

The 4th Pan-American Thalassaemia Conference took place in SĂŁo Paulo, Brazil at the Maksoud Plaza Hotel and Conference Centre on 25-26 March, 2011. The event attracted around 60 participants, mainly physicians, from the Americas and Europe for two days of expert workshops and intensive debate on current treatment and the latest advances in thalassaemia.

Presentations covered transfusion therapy, chelation therapy, emerging challenges for paediatric thalassaemia patients, the kidney in thalassaemia, transfusion and chelation in thalassaemia intermedia, and bone marrow transplantation in thalassaemia. Three breakout workshops focused on heart iron overload, the treatment of children and endocrine disorders.

The Conference also provided an opportunity for participants to learn more about ABRASTA and the experience of patients in Brazil, including presentations on the work of the Thalassaemia Working Group at Brazil's Ministry of Health and a case study of pancreatic iron load in Brazilian patients.

An overview of ABRASTA's work highlighted the organisation's close cooperation with national healthcare professionals and the Ministry of Health. As in many other countries, all three iron chelating medications are currently available in Brazil. In addition, patients enjoy a high standard of transfusion therapy, with safety expected to be further improved with the introduction of molecular testing of individual units of blood for viruses before each transfusion. Viral inactivation will also be introduced when available.

TIF MAGAZINE December 2011

NEWS from our Members Around the World

The country also boasts state-of-the-art facilities for thalassaemia patients, such as those at the Albert Einstein Hospital for the assessment of iron load on the heart, liver and pancreas—essential procedures for all transfusion-dependent patients, including those with thalassaemia intermedia and sickle cell syndrome.

The Conference organisers are extremely grateful to everyone that helped make the event such a success.

Westminster hosts reception

The UK All-Party Parliamentary Group (APPG) on Sickle Cell and Thalassaemia held a reception at the House of Commons on 16 November 2010, entitled Sickle Cell Disease and Thalassaemia: What Next?

The event was hosted by APPG Chair Diane Abbot, MP, with around 100 guests attending, including patients, clinicians, nurses and MPs. The aim of the event was to ensure that the National Health Service (NHS) maintained a focus on thalassaemia and sickle cell patients in the midst of fundamental changes to the NHS. Guests were invited to sign a petition calling for ethnic minorities affected by these diseases continue to receive equal access to NHS services and that funding for the specialised services they require receive adequate funding.

The petition was delivered to the Prime Minister's residence, 10 Downing Street, by representatives of the UK Thalassaemia Society and the Sickle Cell Society, accompanied by MP David Burrowes, on 2 February 2011.

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TIF MAGAZINE December 2011

NEWS from our Members Around the World Cyprus Institute of Neurology and Genetics celebrates 20 years

‘The Cyprus Institute of Neurology and Genetics stands out like a beacon in the Mediterranean Sea attracting the admiration of Europe and the world.’ So said Richard Hughes, President of the European Federation of Neurological Societies, on the occasion of the Institute's anniversary.

Known by its acronym CING, the Institute was founded by Dr Lefkos Middleton in Nicosia in 1990. The scientific and academic communities of the island were extremely underdeveloped at the time. However, given the immense number of neurological and genetic disorders that occur in the Mediterranean region, it was thought essential to build up local capacity to carry out research on such disorders.

CING has established national programmes for genetic testing, as well as developing means of preventing thalassaemia, Down’s Syndrome, hereditary breast cancer and meningitis, among others. It has collaborated with over 100 academic institutes around the world and has published countless scientific articles in internationally recognised journals. New diagnostic, preventative and national programs are currently being introduced, while the academic character of the institute has been augmented with the establishment of a Masters degree in Genetics at the University of Cyprus, from September 2010, with the result that the Institute is now recognised as one of the leading scientific foundations in Europe.

Foundation Against Thalassaemia Faridabad, India The Foundation Against Thalassaemia organised a huge awareness-raising event on 5 June 2011 at Gopal Garden, Faridabad, attracting around 5,000 people for a funpacked occasion that was broadcast live by Divya TV. The day could not have been the success it was without the generous support of a number of individuals. The Foundation Against Thalassaemia wishes to extend special thanks to singer Narinder Chanchal, who made a donation of Rs 50,000 in support of local children with thalassaemia. The event venue was provided by Sant Gopal Gupta and family, who also provided refreshments and dinner for all. The Member of the State Legislative Assembly (MLA) for Faridabad, Anand Kaushik, and Mayor Ashok Arora also pledged to lend political support to patients with thalassaemia.

There is currently no government funding provided for the treatment of children with thalassaemia in India, and local patients rely on our organisation for essential drugs. An additional aim of the occasion was therefore to raise funds for this purpose, as well as to appeal for regular blood donors. Contributions towards the purchase of medicines were gratefully received from H K Batra, Mukesh Aggarwal, Sanjay Kakkar, Manohar Puniyani, I J Kalia and Rachna Sharma.

Kerala patients protest against empty promises

CING's Department of Molecular Genetics, Thalassaemia, has produced outstanding work in developing a new technique for the diagnosis of thalassaemia and other haemoglobinopathies, using Preimplantation Genetic Diagnosis (PGD) and the microarray ‘ThalassoChip’. The development of another innovative technique is currently underway, for the non-invasive pre-natal diagnosis of haemoglobinopathies, via testing of the maternal plasma.

Despite the current economic crisis, Dr Middleton insisted that the Institute’s research must continue to be funded, as a relatively small investment that promises to continue making major contributions to science.

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The Blood Patients Protection Council (BPPC) organised a march on 8 April 2011, in protest at the continued failure of political parties in the southern Indian state of Kerala

to honour commitments to patients suffering from blood disorders. Around 30 protesters were detained by police and subsequently released on bail.

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NEWS from our Members Around the World

Patients in Kerala have been promised that a properly equipped haematology-oncology department would be established at the Kozhikkode Medical College Hospital and the facility upgraded to a centre of excellence, and that patients would be provided with free treatment—pledges that had persuaded BPPC to withdraw its candidate for state Assembly elections, Girija Krishnadas. Following the protest, the state government agreed to provide thalassaemia patients with leuckocyte filters, free of charge.

The march was led by BPPC's Kareem Karassery, along with K S Praseeda, K K Nizar and Sathyan, and included patients with thalassaemia, haemophilia, sickle cell anaemia and leukaemia and their families.

A second day of action was organised to mark World Sickle Disease Day, 19 June 2011, when BPPC observed a one-day fast to draw attention to the needs of patients suffering from blood-related diseases such as sickle cell anaemia, thalassaemia, haemophilia and leukaemia. The fast was led by BPPC General Convener Kareem Karassery and M Muhammed Ershad, the parent of a child with sickle cell disease.

BPPC is demanding that patients with acute blood-related diseases receive a monthly pension of Rs 3,000, as well as compensation for medical negligence. The Council also submitted a list of 11 adolescent sickle cell patients who have died over recent months as a result of inadequate expert treatment. A 20-year-old who died on World Sickle Cell Disease Day was also included in the list. A copy of the memorandum was submitted to Kerala's Chief Minister, the Health Minister and the leader of the opposition.

Thalassaemia Jagruti Foundation An audience of some 250 parents and patients with thalassaemia and cerebral palsy gathered at the Polio Foundation in Ahmedambad on 19 June 2011, for a fun-filled day coorganised with Prathama Blood Centre, the Civil Hospital, Ananya and the Thalassaemia Jagruti Foundation.

The event, hosted by TV personality Hridaynath Garekhan, opened with a performance by Ganesh Stuti of Manali Dance Academy, followed by a musical programme led by Bipinbhai Solanki and a skating performance by Sahil Piyushbhai Shah, a young—and very talented—thalassaemia patient. There was also a fascinat-

ing presentation by Dr Anil Khatri on the Practical Management of Thalassaemia.

The event was marked by a number of examples of exceptional generosity, for which organisers, patients and their families would all wish to give their warmest thanks.

One highlight was the announcement by Dr K M Maheria, Head of Paediatrics at Civil Hospital, that all thalassaemia patients would receive free iron chelation therapy, representing a monthly outlay in the region of Rs 3,000 per patient.

Local chartered accountant Shri Rajnibhai and Hardik Book Store announced plans to provide free text books to children with thalassaemia, while an anonymous donor offered to pay their school fees.

A number of donations were also received: the Thalassaemia Jagruti Foundation received Rs 10,000 from Doliben, while the Polio Foundation and Prathama Blood Centre also received generous financial contributions.

After light refreshments, provided by Ananya, children and their parents were treated to the services of a professional photographer, also courtesy of Ananya, with framed pictures to be presented free of charge to each family.

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TIF MAGAZINE December 2011

NEWS from our Members Around the World ‘The New Generation’ 16th Thalassaemia Camp Malaysia 10-12 June 2011

from complications associated with iron overload because of the challenges of fully adhering to prescribed treatment—a point reinforced by Abu Mansor Bin Muhamad, who also stressed the importance of patients attending regular medical appointments. And he emphasised the extent to which thalassaemics can lead long and productive lives, successfully pursuing further education, fulfilling careers, marrying and having a family.

Another speaker, Benita Aryyani, expressed her gratitude for the moral support she receives, helping her enjoy daily activities just like any other teenager.

The last day of the camp focused on areas such as the role of nutrition, as well as the personal experiences of a number of patients.

More Blood. More life

The 16th Thalassaemia Camp was held in Taiping, Perak, Malaysia, on 10-12 June 2011, attracting more than 100 participants including patients and their families and medical practitioners.

The theme of the event was ‘The New Generation’.

Presentations by patients on living with thalassaemia were extremely well received, as well as talks by medical professionals on issues such as blood transfusion and iron chelation therapy.

Compliance was another important area discussed.

Charendee Soon highlighted the tragic fact that despite free treatment, thalassaemia patients continued to die

World Blood Donor Day, celebrated on 14 June 2011, is an integral part of national blood donor programmes, serving as an opportunity to thank blood donors and to promote voluntary blood donation as a means of securing a safe supply of blood and blood products.

The organisers of the annual event (the WHO, the International Federation of Red Cross, Red Crescent Societies, the International Federation of Blood Donor Organisations and the International Society of Blood Transfusion), invited participant countries to ‘Paint the world red’ by covering or lighting monuments, buildings and popular landmarks with the colour red, and by staging cultural events with a red-coloured theme.

This year’s host for World Blood Donor Day was Buenos Aires, Argentina, which organised events around the theme ‘More Blood. More Life'—highlighting the urgent need for more blood donors everywhere.

Australia

Sydney IVF (now renamed Genea) hosted an event on Preimplantation Genetic Diagnosis (PGD) on 22 March 2011. The evening served to inform patients about PGD, explaining how IVF is used in combination with PGD to help people with genetic disorders have healthy children.

Ali Taher receives honorary distinction Dr Ali Taher, Professor of Internal Medicine at the American University of Beirut (AUB), has been elected a Fellow of the Royal College of Physicians (FRCP) of London. The title of FRCP serves as recognition by one of the most prestigious associations in medicine, and is a great honour for Dr Taher and the Faculty of Medicine at AUB. The announcement is a very timely acknowledgment of Dr Taher’s significant scholarly work in the world of haematology research, particularly in the field of thalassaemia intermedia.

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First Lady of Iraq receives award

Iraq’s First Lady, Hero Talabani, was one of the individuals recognised by the Kurdistan Thalassaemia Care Centre for her work helping thalassaemia patients in the Kurdistan region. The award ceremony took place at the Sulaimani office of the Kurdistan Thalassaemia Care Centre.

Pakistan Thalassaemia Welfare Society

TIF MAGAZINE December 2011

NEWS from our Members Around the World

Pan-Hellenic Thalassaemia Conference

The annual Pan-Hellenic Thalassaemia Conference took place on 30 September-2 October 2011 in Ioannina, Greece.

The conference, organised by the Hellenic Thalassaemia Federation, attracted over 650 participants, including patients, medical professionals and medical students. The main objective of the Conference is to provide updates on recent scientific research and other important developments in thalassaemia.

Baku hosts the 61st WHO Regional Committee for Europe

Ministers and representatives from the 53 countries of the WHO European Region met in Baku, Azerbaijan, on 12-15 September 2011, for the 61st session of the WHO Regional Committee for Europe. TIF representative Dr Victor Boulynjekov participated as an observer at the Regional Committee meeting, contributing a presentation on the health agenda for 2012-2020.

Dr Boulynjekov also made a statement entitled ‘Management of Thalassaemia: A multi-organ disease – A public health issue’, calling on the WHO European Regional Office to adopt a more active role in supporting the fight against thalassaemia and other haemoglobinopathies within the WHO's non-communicable and rare diseases programmes.

TIF has stressed the importance of more effective disease control management in European countries, in light of the increasing migration to Europe of populations from affected countries. A further issue highlighted is the difficulties European health systems face in communicating with members of such populations due to language, cultural and social constraints.

Most European countries have no national prevention programmes in place, risking an increase in the number of affected births. TIF has therefore suggested that the WHO European Regional Office assists in the promotion of such national control programmes, as well as in the collection of accurate, up-to-date epidemiological data on the haemoglobinopathies, and in the adoption of guidelines for the consistent management of such disorders across Europe. TIF has also called for the creation of European Reference Centres to support cooperation between patient communities and the medical profession.

The Pakistan Thalassaemia W e l f a r e Society has had a busy few months.

The Society was very grateful to London's Bismillah C h a r i t y Foundation for organising a visit by Dr Farrukh Tasnim Shah, Consultant Haematologist at the Whittington Hospital and University College Hospital, London who came spent the day with us on 27 April 2011. The Foundation also donated five pumps and 600 vials of Desferal.

During her visit Dr Shah held a free clinic for thalassaemia patients, seeing around 50 patients suffering from retarded growth, endocrine deficiencies, and cardiac and hepatic complications. Each was given a detailed examination and treatment advice.

The opportunity to receive such expert assessment was greatly appreciated by patients and parents, as well as by observing medical staff.

The Society also organised a visit to Sozo Adventure Park in Murree for over 40 patients and parents. The visit aimed to raise awareness about thalassaemia, through the distribution of pamphlets and a series of motivational lectures by Shabnum Ijaz and Murtaza Ali Burhani.

On 3 August 2011, the Pakistan Crescent Society held a ceremony to mark the donation of 50 pints of blood to the Pakistan Thalassaemia Welfare Society. Lt-Gen M Kamal Akbar, Vice President of the Society, received the generous donation with gratitude.

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TIF MAGAZINE December 2011

TIF REGIONAL Conferences

3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias 24-26 October 2012 / Limassol, Cyprus Topics for discussion include: health inequalities across the EU; cardiac, endocrine and liver complications; and advances in the care and cure of haemoglobin disorders and rare anaemias. The programme includes a day of policy-related discussion on control, prevention and clinical management, the conclusions of which will be submitted to the European Commissioner for Health and Consumers Affairs.

Patients, organisations, healthcare professionals, academics and policy-makers from all levels (national, regional and EU) are encouraged to attend.

Thalassaemia sets the pace TIF is delighted to announce the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias, to be held in Limassol, Cyprus on 24-26 October 2012.

The Conference will be held under the auspices of the Cyprus Presidency of the European Union (EU) and the Cyprus Ministry of Health, and is expected to attract participants from all 27 EU-member states.

The overall theme of the event is the growing public health burden of chronic and rare diseases in Europe, with a particular focus on patient rights and policy-making.

The Cyprus Presidency of the EU Cyprus assumes the Presidency of the Council of the European Union in the second half of 2012, offering an invaluable opportunity to highlight issues of concern across the Union. At a time when societies across Europe face considerable challenges, Cyprus is determined to use its Presidency to promote principles of solidarity, humanity and the vision of a healthier Europe by 2020. TIF is delighted to enjoy the full support of the government of Cyprus in playing its part, ensuring that the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias is a success—not just for patients with thalassaemia, but for all those fighting the haemoglobinopathies and other rare diseases.

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This Conference aims to highlight a critical link between research and policy that has led to major breakthroughs in the treatment and prevention of thalassaemia—a trend that TIF strongly believes can be replicated in tackling other haemoglobinopathies. Once a fatal disease, thalassaemia is now a treatable condition; with appropriate national control programmes, it is also preventable.

After years on the fringes, haemoglobin disorders have finally been recognised as a major policy focus; and as an immense burden on national heath systems, requiring policy-makers' sustained attention. The World Health Organisation (WHO) has adopted two specific resolutions on sickle cell anaemia, thalassaemia and other haemoglobinopathies, calling on member states to implement national control programmes as a matter of priority, including training health professionals, promoting community education and cooperating with the WHO to establish expert groups in each region. Other resolutions urge member states to ensure the sustainability of blood supplies, to raise awareness of birth defects (including haemoglobin disorders) and to improve epidemiological surveillance systems. These should serve as the foundation for further improvements in the prevention and clinical care of haemoglobinopathies.

The EU boasts some of the world's highest quality public health services. However, there is considerable variation

Join us in sunny Limassol!

TIF MAGAZINE December 2011

TIF REGIONAL Conferences

The town of Limassol is a leading tourist resort with state-of-the-art conference facilities, set along a 16-km beach front overlooking the Mediterranean Sea. The town is a traveller's delight of historical monuments, from ancient Amathus to a Venetian castle, with soul-stirring stories to match (the marriage of King Richard the Lionheart to Princess Berengaria is just one). All of which promise to make the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias an unforgettable experience.

in standards of management of haemoglobin disorders. It is therefore essential that the EU adopts WHO resolutions in this area, with a view to standardising the control, management and treatment of haemoglobin disorders across the Union. It is also essential that interested parties work together to ensure that the haemoglobinopathies—both malignant and nonmalignant—are presented as a united and distinct group of diseases, worthy of top-class research. To this end, the Conference aims to highlight the immense contribution research into thalassaemia has made to the broader field, particularly in the areas of gene therapy, pre-natal screening and the development of more effective drugs.

The Conference will also focus on the challenges national health services face in the treatment and monitoring of chronic conditions, as well the challenges faced by patients in negotiating highly heterogeneous health systems with great variations in knowledge and expertise between (and sometimes within) countries and regions.

Discussions will include the revision of the Clinical Trials Directive, counterfeit medicines, cross-border healthcare and pharmacovigilance.

Participants The Conference is being organised in cooperation with the Cyprus Ministry of Health, under the auspices of President of Cyprus Mr Demetris Christofias, the Cyprus and European Haematology Societies, and the office of the Cyprus Presidency of the EU. It will be co-chaired by the Minister of Health, Dr Stavros Malas, and TIF President Panos Englezos.

President Christofias has been invited to open the Conference, joined by representatives from the European Commission, the European Parliament, national health authorities and European and international patient organisations. The event is also being supported by the Cyprus Alliance for Rare Disorders (CARD), of which TIF is a founding member, and the EU's ENERCA project.

Over 800 individuals from more than 35 countries are expected to attend, including patients and their families, academics, researchers, health professionals, carers, and public health officials.

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8th of May This year's World Thalassaemia Day was celebrated with all the joy and enthusiasm we have to expect. The slogan for the 2011 event was Equal Chance to Life, inspiring patients everywhere to keep up the fight for fair access to healthcare, education, employment—in short, to fulfil their dreams.

The seminar focused on health policy and governance, as well as marking the official launch of TIF's 'Guidelines for the Clinical Management of Thalassaemia', published by BTF for distribution in Bangladesh.

BTF Secretary-General Dr Md Abdur Rahim provided an overview of thalassaemia in Bangladesh, which has about 350,000 thalassaemia patients. Around 7% of the total population are carriers of thalassaemia and more than 7,000 affected babies are born each year. A major challenge is therefore posed by the inter-marriage of carriers. Thalassaemia has been included in the 2011-16 Health, Population and Nutrition Section Strategic Plan (HPNSSP), however, Dr Abdur Rahim urged officials to ensure that the disease remains high on the policy agenda.

Dr Ali offered his assurance that he would appraise the prime minister of the continued need for diligence in the prevention and treatment of thalassaemia, encouraging the Health Education Bureau to increase awareness of the disease.

TIF MAGAZINE December 2011

World Thalassaemia Day

But Shabab Haider Siddique, a thalassaemia patient, had a wider message, telling the audience that “An equal chance to life does not mean merely living with treatment, but equal rights in education, work and other services.”

Bangladesh Thalassaemia Samity (Society)

Bangladesh Bangladesh Thalassaemia Foundation (BTF)

The Bangladesh Thalassaemia Foundation (BTF) was proud to welcome Prof Dr Syed Modasser Ali, Health Adviser to Prime Minister Sheikh Hasina, as chief guest at a seminar held to mark World Thalassaemia Day 2011. The event was also attended by the Secretary-General of the Bangladesh Medical Association, Prof Sarfuddin Ahmed, the General Secretary of Shadhinota Chikitsak Porisod, Prof Iqbal Arsnal, and the Director of the Bangladesh Medical Research Council, Prof Habib-e-Millat.

The Bangladesh Thalassaemia Samity (BTS) marked the 8th of May with an event at the National Press Club, addressed by the Minister for Social Welfare, Enamul Haque Mostafa Shaheed.

A discussion panel brought thalassaemia patients and their families together with paediatricians, haematologists and other medical specialists, while BTS Vice-President Nazrul Islam made a presentation on the prevention and treatment of thalassaemia.

A number of speakers called for urgent action to improve the care of thalassaemia patients, including:

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TIF MAGAZINE December 2011

World Thalassaemia Day • establishing facilities for the treatment of thalassaemia in all government hospitals • ensuring the availability of blood for transfusions • improving/increasing facilities for the screening of blood before transfusions

Laboratory Officer at the Nicosia General Hospital Blood Bank; and TIF Executive Director Dr Androulla Eleftheriou.

India Foundation Against Thalassaemia The Foundation Against Thalassaemia organised a grand function at DAV Centenary College, Faridabad, hosted by Foundation President RD Sharma and inaugurated by DAV Principal Dr Satish Ahuja. The event was dedicated to the late Gopal Sharma, founder of MVN Institutions and a leading figure in the establishment of the Foundation.

Faridabad Mayor Ashok Arora announced his intention to meet the Chief Minister of the state of Haryana, to request a grant towards the treatment of thalassaemia, as part of efforts to improve patient quality of life.

The Minister responded by noting his deep concern at the increased number of thalassaemia patients diagnosed in Bangladesh each year, acknowledging that government authorities needed to do more to contain the disease and to ensure appropriate patient care. And he invited the submission of proposals for projects to that end, to be funded by the Ministry of Social Welfare.

World Thalassaemia Day was also observed in all 12 medical colleges across the country, following cooperation between BTS and the Medicine Club, a social welfare organisation.

Cyprus The Cyprus Thalassaemia Association in cooperation with TIF organised a workshop entitled ‘Blood Transfusion Therapy—Standards, Quality and GCP (Good Clinical Practice)’, held at the Cyprus Institute of Neurology and Genetics in Nicosia, under the auspices of the Cyprus Ministry of Health. The aim of the workshop was to educate patients and health professionals on European standards in transfusion therapy.

Guests included world-renowned specialists Prof Umberto Rossi, President of the European School of Transfusion Medicine (ESTM), and Niels Mikkelsen, Honorable President of the International Federation of Blood Donor Organisations (FBDO/FIODS).

Speakers included Dr Shubha Allard, Consultant Haematologist at Barts & The London NHS Trust/NHS Blood and Transplant; Dr Alan Kitchen, Head of NTMRL at NHS Blood and Transplant, London; Carolina Stylianou,

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Guests were treated to a much-appreciated song and dance performance by children with thalassaemia—a credit to the work of music teacher Anju Munjal and Ganesha Dance Academy.

Thanks to Mukesh and Shashi Aggarwarl, Mr Kushal and Mrs Palak for their generous gifts, which were greatly appreciated by the performers, as well as to TV personality Nikuj Malik. The event was generously supported

by Venus Industries and Sanjay Kakkar, including a dinner for patients, families and friends.

Blood Patients Protection Council

The Blood Patients Protection Council (BPPC), based in Kozhikode, Kerala, distributed pamphlets, as well as organising a series of discussions for patients and their families. Topics included the prevention of thalassaemia, and the prevalence of depression in adolescent thalassaemia patients.

Dr S P Foundation

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World Thalassaemia Day

ciation websites and holding Nowruz (Persian New Year) exhibitions, as well as cooperating with the ministry of health to distribute information regarding thalassaemia.

Another project highlighted was the purchase of land for the construction of a thalassaemia clinic. But perhaps the most important development of all is the distribution of free oral chelating drugs, which has had a great impact on patients' quality of life.

Dr S P Foundation focused its event on the problem of stigmatisation of patients and the importance of social support for patients. Free blood-tests were available, along with informative talks on prenatal diagnosis.

Awareness-raising pamphlets were also distributed.

Iran

The event included live music and theatre performances, along with face-painting for children. A particularly special moment was taken to recognise the achievements of university graduates and athletes with thalassaemia, as well as those who have overcome the odds to find a happy marriage.

Lebanon Middle East Medical Assembly

Khouzestan Thalassaemia Society The celebration of International Thalassaemia Day in Khouzestan province, Iran, was marked by an event at Aftab Hall, attended by around 500 patients, families, and medical specialists. The event was also attended by members of the Sports Committee of Special Diseases, which works to increase the participation of patients with thalassaemia, diabetes and haemophilia, and organ transplant patients.

The head of the Khouzestan Thalassaemia Society, Dr Forouzan Sadeghian, opened proceedings with a discussion of the Society's activities for 2011, which include seminars on thalassaemia and alloimmunisation treatment, a patient workshop, and free bone densitometry assessments for patients in the province. The society has also provided assistance in setting up thalassaemia asso-

In honour of World Thalassaemia Day, this year's 44th annual Middle East Medical Assembly (MEMA) included a special day of lectures on the haemoglobinopathies. The Assembly, held at the American University of Beirut Medical Centre on 5-8 May 2011, was jointly sponsored by the Cleveland Clinic Foundation and was attended by

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TIF MAGAZINE December 2011

World Thalassaemia Day The event offered a combination of fun and practical advice. Free assessments of bone density were on offer— as well as an abundance of ice-cream and cake. Attractive gifts of a bag printed with the words 'World Thalassaemia Day 2011' were also well received.

There was also time for a highly informative talk on the "Optimal Management of beta-Thalassaemia intermedia" (based on British Journal of Haematology article by Taher, Musallam, Cappellini and Weatherall, 152, 512523), leading to a lively discussion amongst the haematologists present.

Pakistan Thalassaemia Society Of Pakistan around 140 participants with an interest in cardiology, childhood oncology, haematology, surgery and molecular medicine.

There were five lectures on issues related to thalassaemia, including the Khalil Abou Faysal Memorial Lecture, presented by Dr Paul Hassoun of Johns Hopkins Medical Centre, and presentations by Prof Maria Domenica Cappellini of the University of Milan, Dr John Wood of the Children’s Hospital of Los Angeles, Prof Ali Taher and Prof George Atweh of the University of Cincinnati. The symposium was followed by a Q&A session.

Myanmar

Myanmar celebrated World Thalassaemia Day for the first time this year, in an event marked by more than 200 patients and parents.

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The Thalassaemia Society of Pakistan (TSP) celebrated the 8th of May with a colourful variety programme, held at a children's library. The event marked the culmination of a month of activities, including seminars, blood donor drives, awareness-raising lectures and the distribution of informative literature.

Zakia Shahnawz, adviser to Punjab Chief Minister and guest of honour, pledged the support of the Chief Minister's office and applauded the continued efforts of patients and Society staff. She then joined the audience for a warmly-received performance by the children.

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World Thalassaemia Day

Palestine Palestine Avenir Foundation

TSP President Dr Jovaria Mannan highlighted the challenges posed by thalassaemia in Pakistan, where 9,000 afflicted children are born each year and 6% of the population are carriers—a situation aggravated by the custom of marriage between cousins.

TSP General-Secretary Prof Yasmin Raashid stressed the role of prevention, calling for couples to be tested before marriage and for prenatal diagnosis for carriers in the 12th week of pregnancy.

TSP Chief Administrator Abdul Munim Khan appealed for a nationwide push to increase blood donation and to ensure blood safety, warning that patient health was being jeopardised by blood from unauthorised sources. He also said that a great many patients still lacked regular iron chelation therapy.

The Palestine Avenir Foundation/Hippocrates Centre organised a wonderful event to celebrate two important occasion—Thalassaemia and Haemophilia International Days. The event was attended by some 450 patients, parents, official guests and supporters.

Philippines Balikatang Thalassaemia Foundation Balikatang Thalassaemia Foundation marked its 6th annual celebration of World Thalassaemia Day with a conference on the Economics of Thalassaemia, held on 14 May 2011 at Dr Fe del Mundo Medical Centre. The event brought together patients, friends and families, along with staff from Philhealth, the Philippine Charity Sweepstakes Office and the Philippine National Red Cross.

Guests were invited to wear red, and enjoy a day of learning, fun and fellowship, as well as raffle prizes and other surprises.

Palestine Avenir Foundation

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FEATURED Articles

FOCUS: THALASSAEMIA & EMPLOYMENT

TIF Statement on the Employment Rights of Thalassaemia Patients

‘‘

Never allow thalassaemia to hinder your road to success. I take it as a challenge and not a problem, a different ability rather than a disability—one that comes with deeper insights into the possibilities of what life may bring to us. Disease is inevitable. But whether or not it causes ‘dis-ease’ is entirely optional, just as pain is inevitable but suffering is altogether optional.

”

Sukhsohit Singh (patient with β-thalassaemia major, India)

Introduction:

The preparation of this statement was inspired by the experience of Sukhsohit Singh, a thalassaemia patient in India who was rejected for employment by the Indian Civil Service (ICS) on the basis of a Medical Board decision focused exclusively on the fact that he had thalassaemia major—despite his presenting no debilitating complications associated with the condition. Mr Singh has an excellent academic record and performed brilliantly on the ICS exam. The case follows that of Dr Anjali Sardana, also in India, who was rejected for an academic post on the same grounds.

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Current Global Situation: The problem of employment discrimination against patients with thalassaemia and other chronic disorders is by no means confined to India. Indeed, patients in India are in many ways better supported than those in many other countries. As a result of sustained advocacy work by local patients’ associations and specialist physicians, both of the above-mentioned cases have been reconsidered: Dr Sardana is now an Assistant Professor, and the ICS is reviewing the ruling regarding Mr Singh.

Consider the startling results of a recent survey conducted by TIF as part of the ENERCA project on rare

anaemias, funded by the European Commission and involving more than 300 patients over the age of 20 with thalassaemia (90%), sickle cell syndromes (8.5%) and other congenital anaemias. The survey found that a total of 30.7% were unemployed (13.9% through choice), with a further 19.3% working part-time. Only half of patients surveyed were fully employed,.

Europe has robust health and social services resulting in the best survival rates for thalassaemia, and takes a lead role in declarations and conventions on patients’ rights, the rights of the disabled and the chronically sick. But the continent can only employ 50% of the adult affected population. What, then, is to be expected of lower resourced parts of the world?

Another TIF survey, conducted in the Middle East in 2009 and involving 96 patients, found that over 80% felt healthy and strong enough to undertake a full-time job. Despite their desire to work, however, many reported obstacles—from prospective employers and society in general. Less than 20% of respondents felt they faced 'no problem' in getting a job. Just over 30% of patients stated that employers were reluctant to employ people with thalassaemia, while 11.4% stated that they faced outright refusal.

While a few patients (9.4%) cited job shortages as a contributing factor, others referred to prospective employers' own biases. The following are examples of the reactions some job-seekers faced ‘They think it (thalassaemia) is catching’ ‘Repeated absences’ ‘Fear that something is going to happen because of the disease’ ‘They think that we can’t do the job’ ‘Short height’ ‘Difficult to get a job if features, facial or skin colour are obvious’ ‘They see us as parasites’ ‘There is pity which is not a good feeling’

Modern treatment has dramatically increased the life expectancy of patients with thalassaemia major, especially those who adhere to treatment regimes. However, a longer life is not a gift unless accompanied by the fulfilment of one's expectations and abilities, in terms of education, employment

and marriage.

The TIF/ENERCA survey of patients in Europe found that 27.5% of 273 respondents were married, with another 7.7% cohabiting and 2.9% divorced. A total of 35 children have been born to those surveyed and 48.6% of 150 respondents were university graduates. All of which only reiterates what we already know: it is possible for thalassaemia patients to fulfil their aspirations to be productive members of society, supporting themselves and their families.

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FEATURED Articles

Responsibility of Employers and Governments to Ensure Patients’ Employment: Article 23 of the Universal Declaration of Human Rights, 1984, states, 'Everyone has the right to work, to free

Police Academy: Dreams do come true!

The dream of a lifetime came true for Mohammad Faizan, when he was enrolled to serve as a police inspector for a day (and paid!).

Rawalpindi Highway and Motorway Police offered the position to 18year-old Mohammad, who has thalassaemia, as part on an awareness-raising campaign by the Pakistan Thalassaemia Welfare Society to commemorate World Thalassaemia Day. After a day of patrolling, monitoring speed limits and briefing officials, Mohammad said the experience had given him great hope for what life had to offer.

"I ask all thalassaemics to live happily and fight the disease courageously," he said.

The occasion was no less moving for Mohammad's mother, who confessed that she cried with joy at the sight of her son in a police uniform.

"All I can say is may he live long and become a real officer one day," she said.

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FEATURED Articles choice of employment, to just and favourable conditions of work and to protection against unemployment.'

leading a normal life. And such as argument then raises the following questions:

This document has been further bolstered by the 2006 United Nations Convention on the Rights of Persons with Disabilities, which has been ratified by a number of countries, including India in 2007 and Cyprus in 2011. The Convention binds signatories to meet the needs of persons with disabilities, guaranteeing their full human rights without discrimination and with equal opportunity, including to work and employment. Article 27 states that ‘State parties recognise the right of persons with disabilities to work on an equal basis with others,' prohibiting discrimination on the basis of disability, assuring equal remuneration for work of equal value, and safe and healthy working conditions. Furthermore, state parties must ‘Promote employment opportunities and career advancement for persons with disabilities’.

• Does not the adult Mr Singh prove that modern treatment has changed the disease outcome?

• Given his many years of consistent academic achievement, in the course of which he has bettered the majority of his peers, not prove him eminently suited to performing the duties of a civil servant?

Employers and governments alike should carefully consider the issue of health as a condition of employment.

In addition, medical boards should include specialists on the conditions under consideration. Indeed, the medical communities of many countries include a good number of thalassaemia patients who are themselves medical practitioners.

The Role of Thalassaemia Expert Centres and Medical Centres:

The question, however, is whether a thalassaemia patient Traditionally, a doctor's duty to a patient ends with the is a person with a disability. In the case of Mr Singh, the provision of appropriate medical treatment. In the case Prime Minister of India responded to initiatives by the of chronic conditions, psychosocial issues have National Thalassaemia Association and the wider medical normally been left to others—or to patients community by proposing to ‘include thalassaemia as one of themselves. However, with education, employment and the disabilities covered by the Disability Law, which other social issues increasingly recognised as presumably arises from India’s ratification of the important factors in overall patient Convention on the Rights of well-being, this must change. After persons with disabilities. The The fact that people with all, it is the medical experts who are definition of disability in the most aware of how their treatment Convention is stated in Article thalassaemia are living well can change patient survival and 1 as follows: ‘Persons with into adulthood means that quality of life. It is therefore also disabilities include those who their duty to raise awareness in have long-term physical, older patients now face broader society. It is also their duty mental, intellectual or sensory additional responsibilities in to advocate their patients' interests impairments which in in whatever forums may affect interaction with various terms of taking the initiative patient well being, including barriers (attitudinal or to act as advocates for their educational authorities, potential environmental) may hinder employers and government their full and effective right to fulfil their dreams. services. participation in society on an equal basis with others’.

Above all, however, expert centres should ensure that the treatment they recommend does not itself hinder If the Indian Medical Board, which rejected Mr Singh on the patients’ social integration. A key issue here is time of grounds of thalassaemia, did not regard him as disabled, transfusion. Even the best care providers in Europe i.e. as not having a long-term physical impairment, then it tend to offer transfusions in the morning (62%) or the should not have rejected him. If, on the other hand, they did afternoon (32%), with only 5.3% arranged in the regard him as having an impairment, then their rejection evenings or at weekends. This means that thalassaemia was illegal according to the Disability Law of the country. patients attending school or those who work are more The fact is that Mr Singh's application to the ICS was frequently absent, leaving a negative impression on rejected out of prejudice and ignorance, guided by the teachers or employers. In this way, health services are belief that his health might pose a liability to the Service. at least partly responsible for the social prejudice This is an illogical argument based on the idea that patients face. thalassaemia is a fatal condition that prevents patients

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My Identity as a Thalassaemic My name is Somdutta, and I am a 22-year-old with thalassaemia intermedia. But that is not my identity.

I am from Kolkata, working in Delhi as Deputy Manager in Corporate Communication at one of India's leading foreign trade enterprises. I have been a first-class student all my life, always balancing work and play to the best of my ability, and I share the common desire to lead an independent, productive life by dint of merit and hard work. I expect to go places.

But my experience in finding employment has not all been plain sailing. Despite having had the good fortune to receive a top-class education, I almost missed the chance of a job that I am ideally suited for, because I have thalassaemia.

After graduating from Presidency College, Kolkata with Honours in English Literature, followed by a post-graduate diploma in Advertising and Public Relations from the Indian Institute of Mass Communication, New Delhi, I received several job offers. The one I wanted—a prestigious public sector job—seemed the ticket to everything I hoped to achieve. But there was a catch I had not foreseen.

In the course of a medical assessment, I declared myself to have thalassaemia intermedia. I did so with no reservations, knowing that the condition was of no consequence to my fulfilling my life as a professional. After a long wait, I began calling the office every few days, always receiving the same non-committal answer.

My frustration grew and I found myself putting my life on hold, crawling from one day to the next. The media was full of claims and counter-claims in the case of Sukhsohit Singh (see above), who was fighting for his right to a place in the Uttar Pradesh civil service. I began to despair.

After several months without progress, my parents and I decided to visit the Thalassaemics India office in Delhi.

Suddenly, a much needed support system opened up.

Shobha Tuli immediately took up my case, and her encouragement did much to dispel my sense of hopelessness.

Mrs Tuli contacted my prospective employer, request a face-to-face meeting with the HR Manager. By the end of the week she had had her meeting, followed up by a letter in which she cited the Universal Declaration of Human Rights, emphasising the fact that thalassaemics had the same ability—and right—to work.

The involvement of a credible and heavyweight non-profit third-party acted as a catalyst, resulting in a perceptible change in the attitudes of my prospective employers.

I was asked to meet the company’s Chief Medical Officer, with whom I had a friendly chat, followed by another health check, this time with a specialist a haematooncologist. I was pronounced medically fit, with a recommendation that I receive regular check-ups, and was finally presented with a formal job offer two days after India celebrated 65 years of independence.

With help from the right people, I won my battle. But we are a long way from winning the war. For every person who refuses to give in to defective bureaucracy, there are many without the resources to go on. There is scant knowledge about thalassaemia in Indian society and, as they say, a little knowledge is a dangerous thing.

Discrimination against the condition is rampant, particularly amongst employers. The fact that people with thalassaemia can and should lead normal lives still eludes many. That is is a problem that deserves the immediate attention of policy-makers.

The question of how to bring service provision in line with broader patient needs is a long -standing challenge that will require concerted pressure from adult patient populations and their support associations. TIF's recently established Expert Patients Group should prove a valuable source of help in spreading the message.

patients are not alone in this fight: associations and health providers stand ready to offer support and encouragement, just as their families have done in the past.

The Role of Patients in Changing Attitudes Concerning Employment: The fact that people with thalassaemia are living well into adulthood means that older patients now face additional responsibilities in terms of taking the initiative to act as advocates for their right to fulfil their dreams. However,

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FEATURED Articles

Perhaps this is provocative. Every individual with thalassaemia spends each day fighting. Who has the right to say, do more?

But I know we can do more. Everyone—TIF, national thalassaemia associations, friends and families—can learn more about the legal rights of thalassaemics and work to ensure that these are enforced. Most of all, it is the duty of us as thalassaemics to recognise our duty to ourselves and to society.

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We provide valuable medicines to rare disease patients SOIUKP1110 - Date of Preparation: March 2011

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MEDICAL Focus

Synthetic blood: An end to blood drives?

A new study by researchers in France has demonstrated the clinical feasibility of a stem cell-based alternative to donor blood-derived red blood cells, raising the hope that patients in need of a blood transfusion might one day become their own donors.

The authors of the research, Marie-Catherine Giarratana, Hélène Rouard et al, recently published their findings in Blood, the Journal of the American Society of Hematology.

According to the article, the team successfully generated red blood cells (RBCs) from stem cells, injecting the RBCs back into the same individual. The transfusion, administered to a volunteer, showed that after five days the survival rate of the cultured red blood cells (cRBCs) was 94-100 per cent. At 26 days, the survival rate stood at 41-63 per cent—consistent with a conventional half-life of native red blood cells of 28 days.

This research marks the first time researchers have successfully injected a human donor with cRBCs created from human haematopoietic stem cells—the cells from

which all blood cell types are generated, including RBCs. Annual global demand for donor blood currently stands at 90 million units of RBCs—a figure that rises every year, just as the number of blood donors is falling. At the same time, the risks of transfusion-transmitted disease remains extremely high. These findings are therefore doubly welcome, showing the way towards increasing sources of haemoglobin while dramatically reducing the associated dangers of transfusion.

The reported findings of this research have demonstrated proof of principle. It is now possible to envisage a time when thalassaemia patients could receive ‘younger’ red blood cells with a high HbF content, thus lengthening the time between transfusions and reducing iron overload. In addition, patients would enjoy the benefits of receiving RBCs from a single, ‘clean’, donor, thus possibly avoiding the formation of antibodies, as well as the risk of disease.

Although a great deal more research is needed, this is good news indeed.

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MEDICAL Focus Ali Taher is a Professor of Medicine with a special interest in haemoglobinopathies. A Consultant Haematologist at the Chronic Care Centre, Beirut since 1993, Prof Taher is a leading TIF partner and recently received the Medical Alumni Chapter Gold Award for outstanding contributions to his field. Prof Taher has published hundreds of articles in leading scientific journals, as well as two books and three book chapters. He is co-author of TIF's Guidelines for the Clinical Management of Thalassaemia.

A closer look at β-thalassaemia intermedia Ali Taher, MD and Khaled Musallam, MD

Department of Internal Medicine, American University of Beirut Medical Centre, Beirut; and the Chronic Care Centre, Hazmieh, Lebanon Abstract: Our understanding of the processes underlying the disease process in patients with β-thalassaemia intermedia (TI) has substantially increased over the past couple of decades. There are a number of options available for managing patients with TI, with several studies highlighting the benefits and risks of each approach.

However, until solid evidence-based guidelines are available, individualised treatment should be maintained.

• and genes directly involved in the expression of clinical complications (1).

Significant environmental factors may include malaria infection. The mechanism of disease in TI patients is attributed to three main factors: inability of the bone marrow to produce normal red blood cells (ineffective erythropoiesis); chronic anaemia and destruction of red blood cells (haemolysis); and increased absorption of iron from the gut. Recent work has identified several genetic mutations and internal factors that play a part in regulating these processes, which could be future targets for therapy (2). The combination of these mechanisms leads to several clinical complications in TI, which may not be as frequently observed in patients with βthalassaemia major and which could cause significant morbidity, therefore warranting immediate attention.

β-thalassaemia exhibits across a wide spectrum of clinical severity. At one extreme are patients with a clinically silent, mild anaemia, known as β-thalassaemia minor. At the other are patients with β-thalassaemia major, who present Ineffective erythropoiesis is associated with skeletal severe anaemia from an early age and require regular deformities and osteoporosis, as blood transfusions and iron chelawell as compensatory formation of tion therapy throughout their lives.

Once a diagnosis of TI is masses elsewhere in the body The term β-thalassaemia interme(extramedullary haematopoiesis) dia (TI) refers to those cases that established, the patient that can cause mechanical probfall between these two extremes, should be assessed over a lems. Haemolysis has mainly been and who usually present the need associated with enlargement of the for medical attention later in life. period of several months spleen; however, recent evidence before beginning any form suggests that haemolysis, along The clinical characteristics of TI are of treatment. with other factors, causes a high very heterogeneous, making the frequency of thrombosis in patients understanding and management of with TI and may explain other comthe disease a challenging task. This plications such as pulmonary hypertension (increased article aims to assess significant advances made to that pressure in lung vessels) with secondary right heart failend over the past 20 years. ure. The extra iron absorbed from the gut can accumulate in the liver and (less so) in the heart, as recently observed It is now known that in the case of TI, several genetic and through the application of MRI technology, and may evenenvironmental factors play a part in determining the tually lead to significant liver disease (3). severity of anaemia and clinical complications of the disease (phenotype). Examples of genetic factors modulating The management of patients with TI remains a challenge. the severity of TI are: There are currently no solid, evidence-based guidelines • a broad diversity of mutations affecting the β-globin for management, and treatment relies mainly on constant, genes; expert observation of each individual patient.

• co-inheritance of α-thalassaemia; • several newly discovered genes involved in modifying Once a diagnosis of TI is established, the patient should be the γ-chain response (which alters the production of assessed over a period of several months before beginning foetal haemoglobin);

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any form of treatment. No treatment modality, especially transfusion therapy, should be embarked upon too hastily.

Many patients with TI are unnecessarily subjected to a lifetime of regular blood transfusions, particularly those that present an unusually low level of haemoglobin during a period of infection. In such situations, it is often appropriate to administer transfusions only in the acute case, and not to immediately commit to a longer-term transfusion programme. Moreover, the need for transfusion is dependent not only on the severity of the anaemia but also on the patient’s general well being, particularly with respect to activity, growth, development and the early appearance of skeletal changes or other disease-related complications.

A recent study of 584 patients with TI from the Middle East region and Italy (the Optimal Care study) demonstrated the beneficial role of transfusions in decreasing various complications in TI, such as thrombosis, pulmonary hypertension, heart failure, leg ulcers and extramedullary haematopoiesis (4). The same study also highlighted a higher risk of many disease complications after splenectomy, suggesting that the recommendation of such a procedure in TI requires careful consideration.

The study also showed a beneficial role for iron chelation therapy in TI. However, the efficacy and safety of this treatment is currently being evaluated in a large study using the oral iron chelator, deferasirox. Finally, the promising role played by agents that increase the production of foetal haemoglobin (such as hydroxyurea) has been

documented in several studies, however further research in this area is essential.

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MEDICAL Focus

Although TI is considered a milder form of the disease at initial presentation and diagnosis, TI patients are at risk of developing serious complications, especially as they grow older. Further clinical studies are therefore required, to assess the optimal type and timing of treatment to be offered to this group of patients, to avoid disease-related morbidity.

Key References 1. Sankaran VG, Lettre G, Orkin SH, Hirschhorn JN. Modifier genes in Mendelian disorders: the example of hemoglobin disorders. Ann N Y Acad Sci, 2010;1214(1):47-56.

2. Gardenghi S, Grady RW, Rivella S. Anemia, ineffective erythropoiesis, and hepcidin: interacting factors in abnormal iron metabolism leading to iron overload in β-thalassemia. Hematol Oncol Clin North Am, 2010;24(6):1089-107.

3. Taher A, Hershko C, Cappellini MD. Iron overload in thalassaemia intermedia: reassessment of iron chelation strategies. Br J Haematol, 2009;147(5):634-40.

4. Taher AT, Musallam KM, Karimi M, El-Beshlawy A et al.

Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the Optimal Care Study. Blood, 2010;115(10):1886-92.

Dr Chi Kong Li specialised in Paediatric Haematology/Oncology/BMT at the University of Hong Kong, followed by further training at John Radcliffe Hospital, Oxford, Great Ormond Street Hospital, London, and Fred Hutchison Cancer Research Centre, Seattle. As Vice-chairman of China's Paediatric Haematology Committee, he has a particular interest in childhood leukaemia, haematopoietic stem cell transplantation and thalassaemia. He is Editor of the medical journals Paediatric Blood Cancer and the Chinese Journal of Paediatric Haematology and Oncology, and is actively involved in the development of paediatric haematology in mainland China.

How to select iron chelators in 2012

By Chi Kong Li Vice-chairman, Paediatric Haematology Committee, China

Abstract: With three iron chelators available in many countries, a great number of thalassaemia patients now have a selection of therapies to choose from. For many, the long-sought after option is oral, doing away with the need for invasive injections. However, other aspects of treatment are also important, including socio-economic factors, patient age, severity of iron overload and the organs affected—not to mention availability. New oral iron chelators currently under study offer the hope that such therapy will one day be a reality for all thalassaemia major patients, irrespective of age or income.

The three iron chelators currently available are deferrioxamine, deferiprone and deferasirox. Deferrioxamine, which is administered sub-cutaneously, has the longest history of clinical use, and its efficacy and side effects are well known to clinicians. The oral chelator deferiprone has gained in popularity following its approval by the European Medicines Agency (EMA) and boasts a superior ability to protect the heart, which has been shown to improve survival in many studies. The combined use of deferrioxamine and deferiprone is now recommended by many clinicians for patients who have severe iron over-

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TIF MAGAZINE December 2011

MEDICAL Focus load, especially cardiac overload as demonstrated by MRI. The third chelator, deferasirox, is also oral, providing patients and their doctors with a further alternative for the removal of excess body iron.

In most western countries, the number of new cases of thalassaemia major is decreasing or is maintained at very low incidence. However, in countries lacking comprehensive prenatal screening programmes, the number of new cases born each year remains significant. These transfusion-dependent children usually develop excess body iron and begin chelation therapy at 2-3 years of age.

The standard treatment is the subcutaneous administration of deferrioxamine in the region of the abdomen, demanding a great deal of young patients and of the parents who must come to terms with the difficult task of inserting a needle into their child.

The oral chelator deferasirox can be given to children as young as 2. However, the drug remains prohibitively expensive for most families in developing countries, despite the manufacturer's efforts to reduce costs.

The other oral chelator, deferiprone, is not suitable for children under the age of 5. Previously only available in tablets, deferiprone has recently been introduced in solution form. A six-month clinical study of deferiprone solution administered to 59 children under the age of 6, conducted in Egypt, Malaysia and Indonesia, demonstrated short-term efficacy in reducing serum ferritin. Assuming the same biological efficacy as the tablet form, deferiprone solution certainly benefits young children most, and they may not require injectable iron chelation in the future. However, the long-term effects on young children of deferiprone, including side effects, are still unknown. Two of the 59 children receiving deferiprone solution developed agranulocytosis within the six month period, while 6% developed mild neutropenia. It is not known whether the incidence of agranulocytosis increases with longer duration of deferiprone treatment.1 Joint pain or arthralgia occurred in 4% of patients; one patient experienced severe joint pain that required discontinuation of deferiprone treatment. Whether young children with growing skeletal system will be more prone to joint toxicity is a question that requires further study. The accumulation of more clinical data over a longer period are therefore required before recommending deferiprone as first-line treatment in young children.

A number of studies of older patients over the past 10 years have concentrated on the prevention of cardiac toxicity and thus improved survival. The administration of deferiprone alone or in combination with deferrioxamine has been

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shown to be more effective in removing excess iron from the heart. Deferasirox has also been the subject of careful study regarding cardiac protective effect. A one-year study showed that mild to moderate cardiac iron overload patients benefit more from deferasirox as the T2* measured on cardiac magnetic resonance with better improvement of T2*. Follow-up studies indicated further improvement in cardiac iron status at two and three years.2,3 The process of removing iron from heart cells is slow and it may take several years to achieve very good clearance of heart iron. In patients with severe heart iron overload, as demonstrated by T2* < 6ms, the chance of developing heart failure is 47% in one year.4 Such patients should receive intensive iron chelation to prevent heart failure, with most experts recommending a combination of deferiprone and deferrioxamine. However, studies have shown that about 25% of patients on combination treatment have to stop combined treatment, with agranulocytosis cited as one of the main reasons. The combined uses of deferrioxamine and deferasirox, or deferasirox and deferiprone, have not been subject to full investigation, with the exception of a few individual cases. However, patients are more likely to comply with a treatment of combined oral iron chelators, making it an option that may thus improve survival. Deferiprone has better cardiac protective effect, while deferasirox appears to provide good clearance of liver iron. The combined use of both agents may therefore achieve better overall control of total body iron. Lower doses of either agent used in combination may be possible. However, careful study is required into the optimal recommended dosage of each, and the toxicity profile of combined chelators.

References: El Alfy M, Sari TT, Chan LL; Tricta F, El-Beshlawy A. The Safety, Tolerability, and Efficacy of a Liquid Formulation of Deferiprone in Young Children With Transfusional Iron Overload. Journal of Pediatric Hematology/Oncology.

32(8):601-605, November 2010.

Pennell DJ, Porter JB, Cappellini MD, et al. Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload. Haematologica 2011; 96: 48-54.

Continued Improvement and Normalization of Myocardial T2* in Patients with β-thalassemia Major Treated with Deferasirox (ExjadeŽ) for up to 3 Years. 2010 Annual Meeting of American Society of Hematology, Poster #: III-1055.

Kirk P, Roughton M, Porter JB, et al. Cardiac T2* Magnetic Resonance for Prediction of Cardiac Complications in Thalassemia Major. Circulation 2009;120;1961-1968.

Prof Mehran Karimi Prof Karimi is Professor of Paediatric Haematology-Oncology and Director of the Haematology Research Centre at Shiraz University of Medical Science (SUMS), working in the field of thalassaemia and coagulation. After completing his studies at SUMS in haematology with a focus on thalassaemia and haemophilia, he completed a fellowship at the University of Milan under the supervision of Prof PM Mannucci. He is a member of the International Society on Thrombosis and Haemostasis (ISTH) and has twice been awarded the Razi Festival prize for best researcher, in 2001 and 2008. Prof Karimi has authored over 165 articles and 155 abstracts, and is a regular peer-reviewer for a number of international journals.

TIF MAGAZINE December 2011

MEDICAL Focus

Hydroxyurea management in β-thalassaemia intermedia Dr Mehran Karim Haematology Research Centre, Shiraz University of Medical Sciences, Shiraz, Iran Email: karimim@sums.ac.ir

Abstract: The reported success of hydroxyurea in treating patients with sickle cell disease has led to a focus on its possible benefits for patients with β-thalassaemia major, with studies finding reduced blood transfusion dependency. There are also indications of its beneficial use in the treatment of thalassaemia intermedia.

However, further research is needed into the possible side-effects of the long-term use of hydroxyurea.

Hydroxyurea (HU) is a virtually tasteless, white crystalline powder with the chemical formula of CH4N2O2 (Figure 1).

It is an antimetabolite that can promote foetal haemoglobin (HbF), and is approved by the US FDA for treatment of patients with cancers such as chronic myelocytic leukemia (1). It can also form part of an antiHIV regimen and is sometimes used to treat psoriasis (2,3). Available as a capsule for oral administration, HU is also known as hydroxycarbamide and is marketed under the registered trademarks Hydrea and Droxia.

A dependency on regular blood transfusions and resulting iron overload are key complications of β-thalassaemia major (TM), and reducing the need for transfusions can play an important part in improving the safety and quality of life of TM patients. It has been suggested that the administration of HU in patients with β-thalassaemia major (TM) has beneficial effects to this end, via the mechanism of neutralizing excess α-chains through the production of γ-chains, resulting in partial correction of ineffective erythropoiesis.

Studies of the efficacy of HU in TM have revealed a modest increase in Hb levels, reducing blood transfusion dependency or even altogether eliminating the need for transfusions. One study (2004) showed a good response to HU in transfusion-dependent TM patients in Iran, in whom the Hb level was kept above 9.5 g/dL without transfusion (8). Studies have also shown polymorphism for gG XmnI (gG ã 158 C > T), homozygous for β-globin

Table 1: Results of two newly studies in the efficacy of HU in β-thalassemia major patients.

Results

Length of therapy

HU dose (mg/kg/d)

No of patients

References

20(44.5%) good response with 1.5 gr/dl in Hb level

1 year

16.3±2.3 Raised to 17.4±2.4

45

Bradi et al.

9 good response with Hb level of mean 8.2±0.7 gr/dl and transfusion free

46±25 months

10.9±3

11

Korean et al.

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TIF MAGAZINE December 2011

MEDICAL Focus Table 2: Some studies about the efficacy of HU treatment in β-thalassemia intermedia.

Results

Length of therapy

45.9% transfusion free or ↑Hb>2gr/dl 24.3%↓transfusion to 50% or ↑Hb1-2gr/dl

4-36 months

HU dose (mg/kg/d)

No of patients

References

10-20 (mg/kg/d)

37

Dixit et al.

18 splenectomozed untransfused

Mancuso et al.

6

Gamberini et al.

Mean Hb ↑ 1.5 gr/dl

1 year

5-30 (mg/kg/d)

↓ the size of extra medullarly hematopoiesis mass and cured leg ulcers

3 months

1000 (mg/d)

mutations [IVS-II 1 (G-A) or IVS-I 5 (G > C)], and indicated that α-thalassaemia deletions have a strong influence on the clinical response to HU therapy (6,8). Table 1 shows the results of two new studies regarding the efficacy of HU in TM patients (6,9).

β-thalassaemia intermedia (TI) The term β-thalassaemia intermedia (TI) refers to patients whose clinical phenotype is milder than that of thalassaemia major (TM). The clinical course of TI is characterised by several complications that can be prevented by accurate follow-up.

Despite chronic anaemia, individuals with TI do not require regular transfusion, except in association with concurrent illness (7).

Furthermore, because the α/β-globin Figure 1: Chemical structure of HU imbalance in β-thalassaemia intermedia (TI) is less than that in TM, the use of HU may be (7). The study divided 163 TI patients into two groups, expected to result in better clinical responses and according to blood transfusion dependency. Group I haematological improvements (5), as reported by a consisted of 120 patients receiving regular blood number of studies (6-7). transfusion, and group II consisted of 43 patients with long-interval transfusion or no history of transfusion. All Although many of these studies included only a small patients were treated with 8–12 mg/kg/d of HU number of patients, we reported clinically significant administered orally once a day. responses to HU in a larger group of TI patients from Iran

42

A total of 149 patients (91.4%) showed a positive response to HU therapy, with the mean Hb level in Groups I and II maintained at 9.5 and 9.6 g/dL, respectively. HU was also associated with a marked increased in MCV and MCH. After HU treatment, 97% of patients described an increase in exercise tolerance and sense of well-being. No significant facial changes were observed. Spleen size in non-splenectomised patients remained unchanged in 83% of patients. In addition, HU therapy was shown to decrease complications in cardiac function and to reduce pulmonary hypertension in TI patients (13). Table 2 shows the results of a number of other studies into the effects of HU therapy in TI (10-12).

Another recent study evaluated the adverse effects of low-dose HU (8–12 mg/kg/d) in TI patients who had been treated with HU for a period of 10 years. Most recorded adverse effects were dermatological, neurological or gastrointestinal, and none required the discontinuation of Key references: Katzung B, Masters S, Trevor A. Basic and Clinical Pharmacology, 11th Edition. 2009.

Kumar B, Saraswat A, Kaur I. Rediscovering hydroxyurea: its role in recalcitrant psoriasis. Int J Dermatol.

2001;40(8):530–534.

Biron F, Ponceeau B, Bouhour D, Boibieux A, Verrier B, Peyramond D. Long-term safety and antiretroviralactivity of hydroxyurea and didanosine in HIV-infected patients. J Acquir Immune Defic Syndr. 2000;25(4):329–336.

Charache S, Terrin ML, Moore RD, et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the multicenter study of hydroxyurea in sickle cell anemia. N Engl J Med. 1995;332(20):1317–1322.

Karimi M. Hydroxyurea in the management of thalassemia intermedia. Hemoglobin. 2009;33 Suppl 1:S177-82.

Bradai M, Pissard S, Abad MT, Dechartres A, Ribeil JA, Landais P, et al. Decreased transfusion needs associated with hydroxyurea therapy in Algerian patients with thalassemia major or intermedia. Transfusion. 2007 Oct;47(10):1830-6.

Karimi M, Darzi H, Yavarian M. Hematologic and clinical responses of thalassemia intermedia patients to hydroxyurea during 6 years of therapy in Iran. J Pediatr Hematol Oncol. 2005 Jul;27(7):380-5.

therapy. There were no reports of haematologic toxicity, bone marrow suppression or secondary malignancies during HU treatment (14). These results indicated that low-dose HU therapy in thalassaemia may be well tolerated without serious side effects. However, further research into the possible adverse effects of long-term HU therapy is required.

TIF MAGAZINE December 2011

MEDICAL Focus

The administration of HU has been demonstrated to be effective in increasing Hb levels in TI patients, reducing transfusion dependence as well as the incidence of osteoporosis, extramedullary haematopoiesis, skeletal deformities and splenomegaly, while increasing energy levels. Some studies have also demonstrated positive clinical and haematological responses to HU in TM patients, making it a possible useful alternative to blood transfusion for some patients—one that is inexpensive and delivered orally.

Yavarian M, Karimi M, Bakker E, Harteveld CL, Giordano PC. Response to hydroxyurea treatment in Iranian transfusion-dependent beta-thalassemia patients.

Haematologica. 2004;89(10):1172–1178.

Koren A, Levin C, Dgany O, Kransnov T, Elhasid R, Zalman L, et al. Response to hydroxyurea therapy in betathalassemia. Am J Hematol. 2008; 83(5):366-70.

Dixit A, Chatterjee TC, Mishra P, Choudhry DR, Mahapatra M, Tyagi S, et al. Hydroxyurea in thalassemia intermedia-a promising therapy. Ann Hematol. 2005;84(7):441–6.

Mancuso A, Maggio A, Renda D, Di Marzo R, Rigano P. Treatment with hydroxycarbamide for intermedia thalassaemia: decrease of efficacy in some patients during long-term follow up. Br J Haematol. 2006;133(1):105–6.

Gamberini MR, Fortini M, De Sanctis V. Healing of leg ulcers with hydroxyurea in thalassaemia intermedia patients with associated endocrine complications. Pediatr Endocrinol Rev. 2004;Suppl 2:319–22.

Karimi M, Borzouee M, Mehrabani A, Cohan N. Echocardiographic finding in beta-thalassemia intermedia and major: absence of pulmonary hypertension following hydroxyurea treatment in beta-thalassemia intermedia.

Eur J Haematol. 2009;82(3):213–218.

Karimi M, Cohan N, Moosavizadeh K, Falahi MJ, Haghpanah S. Adverse effects of hydroxyurea in betathalassemia intermedia patients: 10 years' experience.

Pediatr Hematol Oncol. 2010 Apr;27(3):205-11.

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TIF MAGAZINE December 2011

TIF REGIONAL Conferences

1st Pan-Asian Conference on Haemoglobinopathies ROYAL ORCHID SHERATON HOTEL & TOWERS Bangkok, Thailand / 8-10 February 2011

Organised by

Thalassaemia International Federation

44

In collaboration with

ANNOUNCEMENT

DAYone

The first TIF Pan-Asian Conference on Haemoglobinopathies has been postponed, due to catastrophic flooding in Bangkok. Originally scheduled to be held on 3-5 November 2011 at the Royal Orchid Sheraton Hotel and Towers, the Conference will now be held on 8-10 February 2012.

The Conference has been organised by TIF in cooperation with the Thalassaemia Foundation of Thailand, Mahidol University, Thailand and the Thai Ministry of Health.

The first TIF Pan-Asian Conference on Haemoglobinopathies takes as its theme ‘Patients and Health Professionals together for Optimal Care’. The event aims to bring together patients and their families, medical professionals, policy-makers and other interested parties from the South-East Asia and Western-Pacific regions, to exchange expertise regarding best practice in clinical care, patient empowerment and disease prevention. The Conference will also provide an opportunity to assess the status of current control strategies in Asia, and to discuss ways of ensuring that haemoglobin disorders are prominent on national health agendas.

A ceremony in honour of Prof Khunying Soodsarkorn Tuchinda, who is to be presented with a Lifetime Achievement Award, has been organised to coincide with Prof Tuchinda's 90th birthday.

TIF MAGAZINE December 2011

TIF REGIONAL Conferences

8 November 2011ROYAL ORCHID BALLROOM

Inaugural Ceremony: ROYAL ORCHID BALLROOM - 1 Chairpersons: Panos Englezos & Suthat Fucharoen TIME: 16:00 – 19:00 16:00 - 16:10

Welcome Message from the President of the Thalassaemia Foundation of Thailand

16:10 - 16:20

Welcome Message from the President of Mahidol University

16:20 - 16.30

Welcome Message from the Ministry of Health of Thailand

16:30 - 16:40

Welcome Message from the WHO-SEARO/WPRO – Maureen Elisabeth Birmingham

16:40 - 17:00

Welcome Message from the President of the Thalassaemia International Federation – Panos Englezos

COFFEE BREAK: 17:00 – 17:30 TIME:17:30 - 19:00 Chairpersons:

Androulla Eleftheriou & Shobha Tuli

17:30 -18:00

Prof. Sir David Weatherall - Keynote speech ‘The inherited Diseases of Haemoglobin are an emerging Global Health Burden’

18:10 - 18:30

Award Ceremony - Celebrating the 90th Birthday and Life Achievement in Thalassaemia of Prof Khunying Soodsarkorn Tuchinda

18:30 –19:00

Introduction to Sultan Bin Khalifa International Thalassaemia Award

45

DAYtwo

TIF MAGAZINE December 2011

TIF INTERNATIONAL Conferences

9 February 2012

ROYAL ORCHID BALLROOM - 1

TIME: 08:00-09:00 Registration TIME: 09:00-10:00 Session 1 – Country Reports – Current Strategies of Prevention and Management of Haemoglobin Disorders in Asian and South East Asian Country – Country reports Suthat Fucharoen & John Wood Chairpersons: 09:00-09:20 Global Epidemiology of β-, HbE, α-thalassaemia and SCD – Specific Reference to Asia – Vip Viprakasit (Thailand) 09:20-09:40 Overview of Genotypes/Phenotypes of Thalassaemias in Asia – Suthat Fucharoen (Thailand) 09:40-10:00 Prevention programmes for haemoglobinopathies – Appropriate for ASIA – Existing and needed – Policy and Laboratory Perspective – Anuja Premawardhena (Sri Lanka) TIME: 10:00 – 11:30 Session 2 – Current Situation in Control (Prevention & Management) Strategies and Health Systems in Asia Chairpersons: Roshan Colah & Ne Win 10:00-10:10 Representative Ministry of Public Health of Thailand - Thailand 10:10-10:20 Jovarian Mannan - Pakistan 10:20-10:30 Roshan Colah – India 10:30-10:40 Ajit Rayamajhi - Nepal 10:40-10:50 Ransnayake Mudiyanse – Sri Lanka 10:50-11:00 Amalia P. Wahidiyat - Indonesia 11:00–11:10 Farzana Khatoon - Maldives 11:10-11:20 Ne Win – Myanmar 11:20-11:30 Ahmed Waqar Khan - Bangladesh COFFEE BREAK: 11:30-12:00 TIME: 12:00 – 13:00 Session 3 – Current Situation in Control (Prevention & Management) Strategies and Health Systems in Asia Chairpersons: Ahmed Waqar Khan & Hisham Shah Mohammed Ibrahim 12:00-12:10 Chen Ping – People’s Republic of China 12:10-12:20 Vincent Lee - People’s Republic of China – Hong Kong 12:20-12:30 Sourideth Sengchanh – Republic of Laos 12:30-12:40 Hisham Shah B. Mohammed Ibrahim - Malaysia 12:40-12:50 Ernesto d’Y Yuson - Philippines 12:50-13:00 Law Hai Yang - Singapore LUNCH BREAK: 13:00 – 14:30 AFTERNOON SESSIONS:- TIME: 14:30– 17:30 Session 4 – Current situation in Control (Prevention & Management) strategies and Health Systems in Asia 14:30-14:40 Nguyen Anh Tri - Vietnam 14:40-14:50 Piseth Prak Raingsey – Kingdom of Cambodia 14:50-15:00 Sant-Rayn Parischa - Australia 15:00-15:10 Meng Yao Lu - Taiwan 15:10-15:30 Ching-Tien Peng - Genetic screening and prenatal diagnosis of thalassaemias and haemoglobinopathies in Taiwan today COFFEE BREAK: 15:30 – 16:00 Session 5 – OUTCOMES & CONCLUSION Chairpersons: Suthat Fucharoen, Michael Angastiniotis, Androulla Eleftheriou, Panos Englezos, Ramli Yunus & Shobha Tuli TIME: 16:00– 17:30 Interactive Discussion: Outcomes, Conclusions and presentation of strategic plan for Asia (2012-2016)

46

10 February 2012

ROYAL ORCHID BALLROOM - 1

TIF MAGAZINE December 2011

DAYthree

TIF INTERNATIONAL Conferences

TIME: 09:00-17:45 CLINICAL MANAGEMENT Session 6 –

Management and policies: State-of-the-art in the Treatment of:

TIME: 09:40-10:40 Chairpersons:

Miguel Abboud & Elisabeth George

09:00-09:30

β-thalassaemia major – John B. Porter (UK)

09:30-10:00

Non-transfusion dependent Thalassaemias- β-thalassaemia Intermedia and HbE/β-thal types – Elisabeth George (Malaysia)

10:00-10:30

α-thalassaemias -HbH type – Vip Viprakasit (Thailand)

COFFEE BREAK: 10:30 – 11:00 Session 7 –

Management and policies: State-of-the-art in - & WHO Policies:

TIME: 11:00-12.30 Chairpersons:

Vip Viprakasit & John B. Porter

11:00-11:30

The Treatment of Sickle Cell Disease – Miguel Abboud (Lebanon)

11:30-12:00

A multi-disciplinary approach: Centres of Reference – Michael Angastiniotis TIF

12:00:12:30

WHO and Policies on NCDs and Hb Disorders – Androulla Eleftheriou – TIF

LUNCH BREAK: 12:30 – 13:30 Session 8 –

Advances in Curative methods

TIME: 13:30-15:45 Chairpersons:

Philip Leboulch & Khunying Soodsarkorn Tuchinda

13:30-13:45

Gene therapy – Philip Leboulch (USA)

13:45-14:00

Haematopoietic stem-cell transplantation in developing countries – Suradej Hongeng (Thailand)

14:15-14:30

Fetal globin induction in Haemoglobin Disorders – Suthat Fucharoen (Thailand)

14:30-14:45

Understanding the molecular mechanisms leading to depression of γ-globin gene expression – Jim Vadolas (Australia)

14:45-15:00

Copy Drugs/Counterfeit Medicines – Carlo M. Nalin (USA)

15:00-15:15

MRI-based technology for developing countries – John Wood (USA)

15:15-15:30

MRI-based Monitoring tools for Iron Chelation – Pairash Saiviroonporn (Thailand)

15:30-15:45

Stem cell transplantation in Developing countries – BMT Experience in Pakistan – the cost-effective model- Lawrence Faulkner (Italy)/ )/ Naila Yaqub (Pakistan)

COFFEE BREAK: 15:45 – 16:30 TIME: 16:00– 17:15 Session 9 –

FUTURE PROSPECTS

Chairpersons: Suthat Fucharoen, Michael Angastiniotis & Panos Englezos TIME: 16:30– 17:45 16:30-17:00

New advances and future outlook in the management and cure of Haemoglobin disorders – Philippe Leboulch (France/USA)

17:00 – 17:30

Patients as active and valuable partners in achieving progress and advances – Panos Englezos (TIF)

17:30 – 17:45

Role and commitment of National Health Authorities in Asia towards Chronic Genetic diseases – Representative from the Thai Ministry of Health

47

TIF MAGAZINE December 2011

UPCOMING Events EVENTS CALENDAR 2012 Event Workshop for Development of National Guidelines for Sickle Cell Disease EFGCP Annual Conference 2012 - Informed Consent: How Less could be more: Effecting a paradigm shift so we do inform patients

Venue

Date

Nicosia, Cyprus

January 12-13

Brussels, Belgium

January 24-25

Luxembourg, Luxembourg Paris, France

January 26 – 27

Paris, France

February 3-4

EUCERD Winter Meeting ENERCA 6th Executive Meeting ESH - ENERCA Training Course on ‘Diagnosis and Management of very rare anaemias: a challenge’

1st Pan-Asian Conference on Haemoglobinopathies and Rare Anaemias Bangkok, Thailand SCS 3rd Patients Educational Seminar

February 2

February 8-10

London, UK

February 22

Brussels, Belgium

February 29

London, UK

March 17-19

European Conference on Rare Diseases and Orphan Drugs

Brussels, Belgium

May 23-25

2nd World Congress on Controversies in Haematology (COHEM)

Barcelona, Spain

October 4-6

3rd Pan European Conference on Haemoglobinopathies

Limassol, Cyprus

October 24-26

Eurordis Gala Dinner: Celebrating Rare Disease Day 2012 IAPO 5th Global Patients Congress

MSc COURSE IN HAEMOGLOBINOPATHY

Make a difference in your professional life and in the lives of people affected by haemoglobin disorders!

A new e-Msc course launched in 2009 by University College London (UCL) and TIF UCL in one of the most prestigious universities in the world and one of the few to have the necessary infrastructure and expertise to teach through e-learning.

This course represents a unique opportunity for health professionals to specialise in haemoglobinopathies online with minimun disruption to professional and personal lives.

For further information, please visit www.instituteforwomenshealth.ucl.ac.uk or www.thalassaemia.org.cy/msc.html or contact TIF or UCL:

48

It is designed to meet the needs of a wide range of medical professionals, including: • medical graduates interested in haemoglobinopathy (general physician, specialists such as pediatricians, haematologists, clinical geneticists, obstetricians/gynaecologists, behavioural scientists) • Science graduates interested in medical research related to haemoglobinopathy and genetics

• other healthcare professionals interested in haemoglobinopathy (counsellors, clinical psychologists, nurse specialists and midwives)

UNIVERSITY COLLEGE LONDON “MSc in Haemoglobinopathy” 88-96 Chenies Mews London WC1E 6HX, United Kingdom Tel: +44 (0)20 7679 6060 Fax: +44 (0)20 7380 9984 Email:Sc_haemoglobinopathy@ucl.ac.uk

THALASSAEMIA INTERNATIONAL FEDERATION “MSc in Haemoglobinopathy” 31 Ifigenias Str., 2007 Strovolos, Cyprus Tel: +357 22 319 129 Fax: +357 22 314 552 Email: thalassaemia@cytanet.com.cy

SUPPORTED BY:

BOARD MEMBERS Corner

About Thalassaemia International Federation ΤIF

The Thalassaemia International Federation (TIF) is a non-profit, non-govermental organization, founded in 1987 by small group of patients and parents representing mainly National Thalassaemia Associations in Cyprus, Greece, UK, USA and Italy - countries where Thalassaemia was first recognized as an important public health issue and where the first programmes for its control, including prevention and clinical management had started to be promoted and implemented.

MISSION

“Promotion of control programmes and access to quality treatment for every patient with Thalassaemia where ever he or she may live”

OBJECTIVES

Τhe objectives of the Federation in addressing effectively the needs of the world Thalassaemia family have since its establishment remained the same and include: • The establishment of new and promotion of existing National Thalassaemia Patients/Parents Associations • Encouraging, motivatiing and supporting studies and research for furher improving prevention strategies, clinical care and for achieving the long-awaited final cure • Extending the knowledge and experiences gained from countries with successful control programmes to those in need.

JOIN US, become a member of our world thalassaemia family!

Thalassaemia International Federation P.O. Box 28807, Nicosia 2083, Cyprus Tel.: +357 22 319 129 Fax: +357 22 314 552 Email: thalassaemia@cytanet.com.cy • www.thalassaemia.org.cy