3rd Pan-european Conference - Executive Summary

Executive Summary of 3rd Pan-European Conference More than 750 participants from 17 of the 27 Member States of the EU as well as other surrounding countries of the Eastern Mediterranean Region and beyond gathered in Limassol to witness the most successful regional conference on haemoglobinopathies and rare anaemias by far organised by the Thalassaemia International Federation until now.

The 3rd Pan-European Conference targeted to address the needs for further education and update of the medical and patients/parents communities and very importantly to provide a unique forum of interaction, discussion and networking within and between the two communities.

The Conference aimed primarily to cover prevention, management and curative approaches and research in the field of haemoglobinopathies and rare anaemias in Europe, but also many other topics related to health systems, chronic disease models and European policies, directives and regulations related to rare diseases.

Ulrich Jäger and Demetris Michaelides, two academics, one in the medical field (former) and one in the field of archaeology (latter) presented the two keynote speeches prior to the main programme. Professor Jäger guided us through the dramatic advances and breakthroughs that have occurred in recent years in the field of haematology and in particular in molecular diagnostics, targeted therapies and hence the way to personalized medicine. Particular focus was given on the work of EHA towards addressing the need for further scientific programs to be secured through funding on a national, European and international level and for the harmonization of standards and education of the haematologists and generally the medical profession throughout Europe to truly promote and achieve equal access of all Europeans to quality treatment in the field of haematology.

Professor Michaelides, guided the audience through the historical documentation and evidence of medical practice in Cyprus. Since the ancient times of Hippocrates and Galen (1108) extremely important archaeological medical findings including medical equipment, surgical tools and medicines (herbal and mineral related) provided unequivocal proof.

On the first day of the Conference (Thursday 25th October) two programmes ran in parallel – the scientific and the patients/parents, while on the second day (Friday 26th October) further to the continuation of the scientific programme, a number of other sessions ran in parallel covering a

wide variety of topics including sickle cell disease, rare anaemias, reference centres, laboratory aspects and the impact of migration policies on the control of haemoglobin disorders in Europe, just to name a few of the most important.

The scientific programme was ‘launched’ with Professor Dimitris Loukopoulos reviewing the history of the advances that have taken place through the years in the knowledge and the understanding of the pathophysiology and the natural history of β-thalassaemia and that have led to the development of such treatment and monitoring interventions that truly converted a paediatric fatal disease into a chronic one. Erol Baysal and Marina Kleanthous covered the topics of prevention with an overview of the accumulated experience and know-how from successful programmes and with particular reference to new approaches and technical methods.

Prenatal diagnosis by CVS or AF, the case of iPS cells for transplantation in the perinatal period, PGD in diagnosis and as a therapeutic approach and non-invasive prenatal diagnosis were amongst the key topics presented in this session. As in every educational event related to haemoglobin disorders and more particularly thalassaemia, iron constituted a central theme, and this time extended to cover rare anaemias as well. The molecular and cellular basis of the toxicity of iron in overload disorders, the genetic and metabolic effects of iron metabolism in rare anaemias and monitoring of treatment of iron overload were the main topics covered in this session.

Cardiovascular disease, endocrine complications, growth, diabetes, fertility and bone disease part of the multidisciplinary needs of these disorders were comprehensively covered as overviews while focus on new advances was placed on blood safety and pathogen inactivation, liver disease, the diagnosis and monitoring and treatment of chronic hepatitis and on the cardiac and liver monitoring tools and technologies.

Highlights sessions Satellite symposium on mortality – The progress achieved and the demands behind it as well as the remaining gaps and challenges Non-transfusion dependent thalassaemias (NTDT) – The new focus of the medical community based on new information and evidence on how to improve treatment protocols of this group of

haemoglobinopathies characterized by a wide genetic variability and long-term underrecognition particularly in the area of management.

Other important topics covered in the scientific sessions included the Sickle Cell Disease Session and the Rare Anaemias one, which covered the diagnosis, epidemiology and treatment and the many new advances achieved in these areas in recent years.

In the final part of the scientific session, other approaches to treatment including induction of HbF, decrease of ι-chains (improving the balance between ι and β chains) and curative methods including bone marrow transplantation and gene therapy were comprehensively discussed.

The patients session concentrated on topics of interest and concern with their own participation both in the choice and also in the delivery of most of the presentations. The programme focused on bone disease, pain, patient/doctor relationship, disability, fertility in men and women, and liver disease. Focus was also placed on newly, emerging complications including renal and the session followed interactive, productive discussion.

Further to those directly related to medicine and science other topics of importance and relevance to chronic/rare diseases were discussed including patient-centered healthcare systems and effective services, and the development of better and more improved chronic care models. Topics of constant concern to patients with chronic diseases of multi-organ nature and multidisciplinary needs, were also discussed including adherence to treatment and patients as partners in medicine.

Conference Abstract Book The Abstracts from both the Poster Abstract Session of the Conference and the Scientific Programme can be found at http://www.thalassemiareports.org/ .