A Paradigm Shift for Women with Bleeding Disorders

Spring/Summer 2020 I Volume 15

A CHES Publication

A

FT ARADIGM HIfor WOMEN with Bleeding Disorders

Complimentary & Alternative Medicine ...Uses for Pain in Bleeding Disorders l Your Joint Health is Important l Are You or Your Child Feeling Marginalized? Here's Help! l Going Molecular: The Newest and Coolest Bleeding Disorder Treatments l Anxiety is Normal l and more!

Inhibitor-Focused Friday, June. 26th - 29th, 2020 (Fri-Mon) Camp Zeke - Lakewood, PA

Going Vir tua l

After the ShockSM was created to give children with inhibitors the same annual camp opportunities and experiences as their peers in the bleeding disorders community. This extended weekend program is for children ages 0-18, open to the entire, immediate family, and packed full of education, support and – most of all – fun designed specifically for children with active inhibitors and their families. We play, learn, and grow while we build stronger relationships.

Comprehensive Health Education Services has been serving the needs of those with rare bleeding

Oct. 21st - 25th, 2020 (Wed-Sun) YMCA Camp Collins - Gresham, OR

Going Vir tual

LeverageSM is a pioneering, national program for young adults from ages 18+ that have hemophilia with an inhibitor. In partnership with GutMonkey, the program consists of life changing experiences that allow participants to challenge themselves in ways they never thought possible through a series of outdoor experiential education activities.

conditions since 2009.

July 17 - 19 , 2020 (Fri-Sun) Boston, MA - TBD th

SM

As long time members of the bleeding disorder community, our mission is

LEVERAGE

SM

th

Going Vir tual

MomentumSM is a men’s only, national program for those ages 18+ that continue to live with both hemophilia and an inhibitor. The program provides participants with opportunities to not only speak with specialists familiar with inhibitors, but most of all, amongst themselves about issues and challenges important to them.

to inspire awareness and

Rare Disorders

self-reliance for patients with chronic health conditions, their families, and their communities.

More details on our programs can be found on our website:

Sept. 11th - 13th, 2020 (Fri-Sun) Westin Detroit Metropolitan Hotel - Detroit, MI One Drop welcomes individuals and their immediate families living with all rare factor deficiencies (including FVd, FVIId, FXd, & FXIIId), and Glanzmann’s Thrombasthenia. One Drop brings those affected by rare bleeding disorders together to establish a community. This program may offer a voluntary molecular sequencing study to determine individual genetic information with Dr. Diane Nugent of Orange County, CA and the Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders. This study began in 2017. *This program will also be offering the U. of Miami HTC’s joint imaging study.

The Factor 7 Retreat and GT Symposium are now a part of the One Drop program.

www.ches.education

Women-Focused Oct. 23rd - 25th, 2020 (Fri-Sun) Newport News, VA

Comprehensive Health Education Services, LLC 89 East Washington Street Hanson, MA 02341-1125 (781) 878-8561

The LadyBugs program empowers women ages 16+ that are affected by, care for someone, or carry a bleeding disorder.

The program’s goal is to assist women to find their voice when it comes to decision making about the health of themselves and their loved ones. *This program will be offering the U. of Miami HTC’s joint imaging study.

*Women ages 18-50 with any factor deficiency may volunteer for an ultrasound imaging study to assess joint arthropathy under the guidance of Dr. Fernando Corrales-Medina and Dr. Kelli Fraga from the University of Miami HTC under the direction of Dr. Joanna Davis. This study began in 2019.

FACTOR REPLACEMENT REFLECTS THE PROTECTION WITHIN

For people with hemophilia, Factor treatment temporarily replaces what’s missing.1,2 With a long track record of proven results, Factor treatment works with your body’s natural blood clotting process to form a proper clot.2-6 Brought to you by Takeda, dedicated to pursuing advancements in hemophilia for more than 70 years.7

Stay empowered by the possibilities.

References: 1. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197. 2. Canadian Hemophilia Society. Factor replacement therapy. http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/the-treatment-of-hemophilia/ factor-replacement-therapy/. Accessed May 18, 2018. 3. Franchini M, Mannucci PM. The history of hemophilia. Semin Thromb Hemost. 2014;40:571-576. 4. Hvas AM, Sørensen HT, Norengaard L, Christiansen K, Ingerslev J, Sørensen B. Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A. J Thromb Haemost. 2007;5:2408-2414.

5. Antovic A, Mikovic D, Elezovic I, Zabczyk M, Hutenby K, Antovic JP. Improvement of fibrin clot structure after factor VIII injection in haemophilia A patients treated on demand. Thromb Haemost.

2014;111(4):656-661. 6. Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. In: Biochemistry. 5th ed. New York, NY: WH Freeman; 2002. https://www.ncbi. nlm.nih.gov/books/NBK22589/. Accessed May 18, 2018. 7. Shire. Shire’s 70+ year commitment to the hemophilia community. https://www.shire.com/en/newsroom/2018/january/7sossj. Accessed June 6, 2018.

Copyright © 2019 Takeda Pharmaceutical Company Limited. 300 Shire Way, Lexington, MA 02421. 1-800-828-2088.

All rights reserved. TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited. S46132 03/19

Follow us to keep abreast of our programs and announcements facebook.com/ CHES.Programs

Letter From the Editors Welcome! 2020 has brought many changes to our community and to CHES. The first change you will notice is within this publication. We are excited to bring LifeLines for Health to the entire bleeding disorders community that we serve. This includes rare bleeding disorders, women with any bleeding disorder and of course those managing an inhibitor.

We have championed the needs of underserved populations within this community since 2009. LifeLines for Health has been a consistent resource at every event we offer, so it seems like a natural progression to distribute to all of our families.

As we go to print, we are immersed in a global pandemic and quarantine from novel COVID-19.

With so much information flooding the airwaves, it is a challenge to separate fact from conjecture. As individuals living with chronic conditions, many of us find comfort in the science. This community is no stranger to living with uncertainty. In fact, it could be argued that remaining at home, being solitary and waiting for the next shoe to drop (because it is a matter of when, not if) has been our typical way of life. Flexibility, patience and resilience are attributes that come to mind when describing those that live with bleeding disorders.

instagram.com/ CHES_Programs

LifeLines for HealthSM Disclaimers The views and opinions of our writers are not a reflection of Comprehensive Health Education ServicesTM, Inc. (CHES) or its sponsors.

This newsletter is designed to provide a forum for community members to express their views from an open and honest platform. It is meant to provide a sharing of knowledge and experience to help one another. Nothing in this newsletter is meant to replace the advice of your HTC, medical professional team or insurance provider. You are always urged to seek the opinion of a healthcare professional for treatment and your specific insurance provider for information.

We take your privacy very seriously. We would never disclose your personal health information without your express written consent. We would never sell nor make available our secure database to anyone.

Articles and pictures may not be reproduced, published, and/or placed on websites without the express written permission of CHES. In every publication of LifeLines for HealthSM, we will provide links to other websites that are not owned or controlled by CHES or its sponsors. We cannot be responsible for privacy practices of other website owners, nor can we be responsible for the accuracy of the information provided.

Programming in 2020 will look different, for several reasons. nSpiration Foundation will collaborate with CHES to present After the Shock: an Inhibitor Family Camp, One Drop: Rare Bleeding Disorders Consortium (combines F7 Retreat and GT Symposia), LadyBugs, and Leverage.

Resources are limited to fund air travel, which is what makes nSpiration Foundation an important partner. It has the ability as a non-profit to accept donations and submit grants to a wider variety of resources. Whether events will be in person programs or virtual remains to be seen. Like most organizations in the community, we are preparing for both. After the Shock will most likely be a virtual event. Although virtual truly isn’t the same, the need for education and connection does not end because it isn’t potentially responsible to gather together in large groups at this time.

In the meantime, we hope you enjoy this issue of LifeLines for Health filled with resources on women with bleeding disorders, complementary methods to manage pain, mindful ways to deal with anxiety (which is at a crescendo for so many right now), and products new to the market and/or in clinical trials. Our friend, Dr. Gary McClain takes a deeper dive into feelings of marginalization; a reoccurring concern expressed by most women with bleeding disorders and those with rare bleeding disorders.

Check out our Community Chatter section regarding updates to the One Drop and LadyBugs’ programs in 2020.

Be sure to check out our website www.ches.education, like us on Facebook, follow us on Instagram to stay apprised of how we plan to keep you up to date and connected. If you have an idea, comment or suggestion, don’t hesitate to let us know at info@ches.education. We love to hear from you!

In the meantime, eat well, practice good sleep hygiene, move, find ways to safely get fresh air and sunshine. Be safe, be well and we look forward to seeing as soon as we can.

- Janet Brewer & Eric Lowe jbrewer@ches.education

elowe@ches.education

"Fear is often our immediate response to uncertainty. There's nothing wrong with experiencing fear. They key is not to get stuck in it."

-Gabrielle Bernstein

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Integrity, Accuracy, Empathy...

FEATURE 10 I A Paradigm Shift for Women with Bleeding Disorders For centuries, women with bleeding disorders (WWBD's) have been ignored, belittled, ostracized even convinced that their symptoms are conjured up by some psychological barrier. But this last decade has marked a pivotal point in our history. Dr. Robert Sidonio of Emory, unarguably one of WWBD's most recognized advocates, provides an overview of the most prevalent clinics, organizations, research projects, and resources of our time for WWBD's.

CONTENTS COMMUNITY CHATTER

FAMILY MATTERS

4 I LadyBugs: Outstretched Wings are Good for Soaring

28 I Are You or Your Child Feeling Marginalized? Here's Help!

Barb Forss, a woman with a bleeding disorder who was once disregarded by her doctors, moved from shadow to spotlight when she founded LadyBugs. She guides us through the evolution and reprisal of this nationally recognized program for women with bleeding disorders that has become known by many as a strong advocacy resource.

Marginalized? You may not know the term, but chances are you've probably experienced it. Unfortunately, women with bleeding disorders are all too familiar with this concept. If you've been ignored by your doctor, you've been marginalized. Get to the bottom of why this is happening with Dr. Gary McClain, and learn some pointers on how you can be heard.

7 I The Factor 7 Retreat & GT Symposium become... One Drop For nearly a decade, the Factor 7 Retreat has brought advocacy and community to those with FVIId and their families. The Glanzmann's Thrombasthenia Symposia similarly followed in later years. In 2020, the two programs will combine to form One Drop, a rare bleeding disorders consortium. Details on the merge are here.

WHAT’S NEW 20 I Complimentary & Alternative Medicine ...Uses in Pain for Bleeding Disorders Due to highly regulated access to pain medication as a result of the opioid crisis, it might be time to look at other options for pain control. A combination approach called CAM has a wide variety to consider. This is a one-of-four part series.

24 I Your Joint Health Is Important

BLOODLINES 34 I Going Molecular: The Newest and Coolest Bleeding Disorder Treatments Exciting times continue on! Perhaps you've already begun using that shiny, new toy in the medicine cabinet. iPhone SE and Galaxy S20 users - I'm talking to you here. Or, maybe you're holding out to see what's coming next. Whatever your shopping methods are, we've got some new treatments here that you don't want to miss out on.

MIND BODY CONNECTION 38 I Anxiety is Normal Anxiety is here, there, everywhere. It lies in him, her, you, and me. Mindfulness expert, Krystyn Strother normalizes this bothersome emotion before arming us with a simple exercise to manage it.

Ask anyone with severe hemophilia who has surpassed his/her 20's and they'll most likely tell you that it's far better to care for your joints early in life than to repair them after the damage is done. Follow these tips for a healthier set of bones.

CONTENTS

Outstretched

by Barb Forss

s g Win for " “

Y

ou’ll probably never meet another Woman With a Bleeding Disorder (WWBD.) There just aren’t that many of you out there.” These were the words of the hematologist who diagnosed me in 1998 with severe, hereditary, FVIId. At age 47.

Before computers, cellphones, and social media were around, resources and connection opportunities were limited.

After attending a training session by NHF called “Woman to Woman”, I was propelled into

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Good

SOARING

Fortunately for me, the words “no”, “never” and can’t” aren’t an integral part of my vocabulary. So, I was determined to find someone who bled like me, ANYONE, who could give me advice, support, friendship, and most of all, help me feel normal and not so alone.

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the direction I needed to go. I needed the comfort of being with other WWBDs and in 1998 LadyBugs was formed for all women with a bleeding disorder.

Slowly at first, then gaining momentum, LadyBugs gathered in cities in my state, mostly as social opportunities. Luncheons or teas, anyplace we could meet and tell our stories. Those stories were important, because they told us that not only were WWBDs not being properly diagnosed, but there was also a general lack of knowledge about treatment options.

One year, we actually had our very own special “Meet and Greet” session at NHF. How amazing to finally be able to give LadyBug Hugs to those I’d only written to or spoken with on the phone! We all felt like long-lost sisters thinking, finally...here’s somebody who “gets it and gets me”. Tears of joy were often mixed with tears of compassion upon hearing each other’s bleeding stories and corresponding treatment or, as was sadly more often the case, a lack thereof.

Spring/Summer 2020

Continued on page 6.

putting our values into action For nearly a century, Novo Nordisk has been a company united for one purpose, helping people. Through our products, our employees, and our community partnerships, we are doing just that.

Please visit novonordisk.us to learn more about how we are moving forward and giving back.

Novo Nordisk is a registered trademark of Novo Nordisk A/S. Š 2017 Novo Nordisk All rights reserved. September 2017

Flash forward almost 22 years, and with the amazing support of CHES, the LadyBugs Program, which started as a grass-roots endeavor to just meet up with others like myself, is now the only program to hold a dedicated annual, national retreat for all WWBDs ages 16 and up. Women who have any bleeding disorder, including carrier mothers, sisters and daughters of men and boys, qualify to be a “LadyBug”.

LadyBugs encourages WWBDs to recognize and advocate for the care they need. Sometimes there are obstacles. That’s why our Retreats are literally loaded with experts from all types of beneficial disciplines. We have sessions with those who are experts at treating WWBDs... hematologists and OB-GYNs, nurses teaching us selfinfusion, and yoga instructors showing us how to lower our shoulders from our ears and b-r-e-a-t-h-e. Mental health experts reminding us it’s ok to say “no” and not feel bad about it. We added an exciting one of a kind study component in 2019, and 2020 for women ages 18-50 with factor deficiencies to participate in the Assessment of Joint Arthropathy Using Ultrasound Technique in Women with Coagulation Disorders (WUP- Women w/ coag disorders Ultrasound Project) facilitated by Fernando Corrales-Medina, MD, and Kelli Fraga, DPT-PI/ Investigators and Joanna Davis, MD, Co-Investigator of the University of Miami.

WWBDs have glowing reports with statements like, “I learned more about my disorder here than ever before!”, “Thank you for empowering me to advocate for myself”, and “I like the intimacy and just women involved. It provides a support system to expand our awareness and education to

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advocate for ourselves and families.” Probably the number one comment we hear at LadyBugs Retreats is, “This is lifechanging for me. I can’t thank you enough.”

The LadyBugs Retreats are making a difference in the lives of hundreds of women living with SHEmophiliaTM, thanks to the generous support of CHES, nSpiration, and the companies who help fund their programs. If you’re interested in learning more about our Retreats, reach out at: https://nSpiration.foundation/ladybugs We also have a closed group on FaceBook where we chat.

And no worries, no one will ever try to sell you a product or service, and your name and stories are protected.

Now don’t forget to give out those LadyBugs Hugs!

Program details such as dates and locations can be found on the inside of the front cover.

The

Factor 7 Retreat and

by Janet Brewer, M.Ed

Glanzmann's Thrombasthenia Symposia

become...

What's with the name?

One Drop refers to the extremely low prevalence of FVIId individuals (said to be 1:500,000), those with GT (1:1,000,000), and others with rare factor deficiencies. Equally important, but small in numbers, hence the name - a drop in the bucket, er pool, er ocean. In parallel, the droplet is a symbol of blood - an obvious choice for these groups.

In 2016, CHES was thrilled to begin a collaboration with Helen Smith, founder of The Glanzmann’s Research Foundation for the first Glanzmann’s Thrombasthenia Symposia. A tireless advocate, Helen created a global community of families affected by this one in a million-bleeding disorder. It was an honor to bring 35 families from across the country together to meet others managing the same challenges that Glanzmann’s brings. Like our Factor 7 families, many had never met another person with GT. We were thrilled to see our families again in 2019 for a second GT Symposia. Dr. Nugent and her team were able to join this group and obtain blood samples for additional research for this ultrarare disorder.

I

n 2012, CHES began serving the needs of those affected by FVIId at a small venue in upstate New York with 18 families in attendance; in 2019, we welcomed 55 families! It was the very first national conference ever held in the US that catered specifically to their needs. As we reflect on the impact this program has had for those with FVII deficiency, the milestones are astounding.

This community has gone from not being sure what a HTC was, or how to self-infuse, or knowing another person with their deficiency, to a robust, empowered family community. The Super Sevens, as they call themselves advocated from the very first Factor 7 Retreat for more research to understand their disorder. In 2017, that call was answered when Dr. Diane Nugent and her team from Children’s Hospital of Orange County, CA attended the retreat to conduct blood samples from every biologically related member of a family for the purpose of identifying molecular variants. The gathering of this information has provided further insights in regard to discrepancies between circulating FVII activity and bleeding symptoms, which often do not correlate.

Our youngest Super Seven!

Hayden Hitch cock,7-month old son of Ah nee Haynes Hitchcock an d John Stephe n Hitchcock rock s his Super Seven shirt!

Great photo Ah nee-thanks for sharing!

Continued on page 9.

COMMUNITY CHATTER

e r o m r e v o c s Di about IXINITY

®

Visit IXINITY.com Aptevo BioTherapeutics LLC, Seattle, WA 98121 © 2019 Aptevo BioTherapeutics LLC. All rights reserved. CM-FIX-0112

IXINITY® [coagulation factor IX (recombinant)] and any and all Aptevo BioTherapeutics LLC brand, product, service and feature names, logos, and slogans are trademarks or registered trademarks of Aptevo BioTherapeutics LLC in the United States and/or other countries.

What do our participant’s say about these programs?

“All the information is current and We informative.”

“We love everything! come; we learn something new to assist with our child’s bleeding disorder.

Kids come together with the same issues and they pick-up friendships exactly where they left off.”

“All of the information I learned was very informative. I gained so much useful information that gives me great hope.”

“Every year I learn something new about Factor 7 and how I can help be a strong advocate for my son. The speakers are amazing.”

“The fact that Dr. Nugent was there and is willing to speak with each of us and explain our reports to us is amazing.

Her research is truly going to help us all.”

So, what is One Drop? In 2020, nSpiration Foundation will collaborate with CHES to combine the Factor 7 Retreat and GT Symposia into one rare bleeding disorder consortium named One Drop. There will be separate in-depth medical sessions for each disorder facilitated by physician experts in these fields. Self-Infusion classes, and sessions on the psychosocial effects of chronic conditions, focus on women’s OB/Gyn needs, and connecting through shared experiences. Teens participate in their own dedicated track that provides time with medical specialists, team building and rap sessions. One Drop will offer two study opportunities this year to include Dr. Nugent’s current molecular sequencing project for all biologically connected family members, as well as The Assessment of Joint Arthropathy Using Ultrasound Technique in Women with Coagulation Disorders (WUP- Women w/ coag disorders Ultrasound Project) for women ages 18-50 with a factor deficiency.

One Drop is scheduled for Sept. 11-13, 2020 in Detroit, Michigan. Due to COVID-19 however, we are closely monitoring and will abide by federal, state, and city guidelines meant to keep everyone safe. Possible contingency plans include postponing to a later 2020 or 2021 date, or a virtual meeting.

Tragically, Helen Smith unexpectedly passed October 20, 2019. The GT community mourns as she was highly regarded and loved by all. CHES sends its deepest condolences to her family. Helen's efforts as founder of The Glanzmann's Research Foundation continue on in her memory. Donations can still be made at: https:// www.gofundme.com/f/glanzmann039s-research

For more info on One Drop, visit nSpiration. foundation/one-drop

COMMUNITY CHATTER

By: Robert Francis Sidonio, Jr., MD, MSc.

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A PARADIGM SHIFT for WOMEN with Bleeding Disorders W omen and Girls clinics

The recognition of the unique challenges of women with bleeding disorders has quickly expanded over the last 2 decades. In 1998, a seminal article was published

by Dr. Peter Kouides highlighting the “silent majority” and setting the stage for the hard work needed to meet these challenges. Back then we knew that it would take a wider medical community to address the needs of women with bleeding disorders (WWBD).

The focus has been to increase awareness amongst healthcare providers focusing first with the hematologists and then other medical providers that are an essential part of a woman’s healthcare including adolescent medicine, gynecology oncology, and primary care providers. Additionally, because of large gaps in care, a multidisciplinary approach would be required. It made sense that the same approach used successfully for men with bleeding disorders, specifically males with hemophilia, would be ideal for WWBD. Modifications of this model were needed of course. This multidisciplinary team that was in place for decades as part of the hemophilia treatment center (HTC) model and established a solid foundation for the creation of the first “women and girls clinics.” Typically, the clinic model employed a hematologist, nurse and gynecologist or adolescent medicine provider. As with the multidisciplinary HTC model, social workers, genetic counselors, and physical therapists are added as available and needed. Most of these women’s clinics are conducted on a recurrent basis, typically once or twice a month. The approach may vary slightly based on hospital or clinic infrastructure. The patient may be seen separately by the hematologist and

FEATURE

gynecologist/adolescent medicine provider or together, my preferred strategy. To make this model work from a financial perspective, the gynecologist or adolescent provider may have a portion of their salary covered utilizing 340B funding, a program created by Congress allowing the HTC to purchase clotting factor concentrates (CFCs) at a discount price with the savings going directly into providing services for the patient, including paying for the salaries of the HTC staff and clinical research initiatives.

Thankfully, more patients are being identified and managed with expertise improving. This is seen in the number of patients registered in various surveillance programs such as the CDC Community Counts and ATHNdataset.

Examples of key data All of these collect surveillance programs: basic demographic On-Going: data and limited information on • CDC Community bleeding events Counts and diagnostic labs • ATHNdataset and in some cases limited information Ended: on quality of life, • Female Universal Data or the affect the Collection bleeding disorder has on the person’s • My Life Our Future life. There are Project some targeted initiatives that have been ended such as Female Universal Data Collection which summarized data on >300 WWBD from 17 HTCs from 2009-2011. Similarly, the My Life our Future Project had a pilot allowing collection of clinical bleeding symptoms and genetic testing in >1000 hemophilia A and B carriers. Ongoing research projects will be discussed later in this article. As a result of more focus on WWBD, the number of women and girls clinics has

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grown from a handful in the early 2000s to >150 in 2020.

These clinics offer a variety of services for WWBD and best practices are currently shared at yearly Learning Action Network meetings sponsored by the Foundation for Women and Girls with Blood Disorders.

Following a few articles in the early to mid 1990s highlighting our lack of ability to address the needs of WWBD, from 1990-2010 there was an exponential rise in the number seen at HTCs at a much higher rate than their male counterparts. It is reassuring that to see the general trend of WWBD being managed by the same top-notch hemophilia providers as their male counterparts.

But are their clinical and research needs being met?

Despite all of the aforementioned progress, there is certainly a gap in the recognition that WWBD may have significant bleeding and a lack of standard practice in the management over their lifetime. The solution to this lies in an improved focus on clinical research in WWBD which over time will translate into improved care and recognition from some reluctant colleagues and payors.

Advocacy Organizations

The Foundation for Women and Girls with Blood Disorders (FWGBD) was launched in 2010 as a non-profit organization with a focus on provider education across all disciplines to ensure women and girls with blood disorders are diagnosed and appropriately managed over their lifetime. This organization has a robust group of mentored young physicians interested in clinical research and innovation in practice and has led to multiple research initiatives. Although the initial focus was on women and girls

with bleeding and clotting disorders, blood disorders such as sickle cell disease, thalassemia, and immune thrombocytopenia are being addressed more over the last 3 years.

While FWGBD focuses on provider education and developing a medical community of dedicated researchers and master clinicians, patient advocacy organizations such as the National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) have dedicated programs to women and girls with bleeding disorders. Victory for Women (victoryforwomen.org) is an NHF initiative created to provide support, education, and resources for women and providers in the bleeding disorders community. The main goal is to address critical issues and ultimately improve quality of life for women in this tightknit community. It recently was rebooted with additional support. HFA has created a Women Bleed Too! Toolkit (hemophiliafed. org) organizing their resources amassed over the years into one location. Acknowledging poor communication

between providers and patients regarding the frequency and volume of heavy periods a smartphone app called Sisterhood (sisterhoodapp.com) was created to empower the patients to track their menstrual periods. Critical to improving awareness and educating the general public is collaboration. HFA and FWGBD created a brochure for providers highlighting the resources available and another brochure targeting the patients at risk for bleeding listing the typical bleeding signs and symptoms of a congenital bleeding disorder.

FEATURE

The NHF and the CDC collaborated on multiple materials focused on WWBD. The CDC has recently updated their materials and information on WWBD (cdc.gov) focusing on recently published research and the impact of a bleeding disorder on the lives of women with bleeding disorders.

Betteryouknow.org is a website for undiagnosed women and men experiencing bleeding symptoms. The campaign was formed to create awareness and provide online resources to improve the time to diagnose a bleeding disorder. The materials are available from the CDC website and directly from betteryouknow.org.

The Canadian Hemophilia Society (hemophilia.ca) has long provided resources for WWBD and Code Rouge was a focused initiative to improve the time to diagnose a bleeding disorder for women with reproductive tract bleeding (heavy menstrual bleeding and perimenopausal bleeding). To

support their mission, they have meetings bringing together healthcare providers to educate, disseminate best practices, and debate controversial topics.

Focusing on the most common bleeding symptom in WWBD, Dr. Paula James (my personal clinical research hero) of Queens University created a website (letstalkperiod. ca) with an online version of bleeding score (ISTH Bleeding Assessment Tool) that allows at risk women to answer a brief bleeding inventory and determine whether they may be at risk for a bleeding disorder such as von Willebrand Disease. This sort of direct to patient initiative empowers women to advocate for themselves and arms them with objective bleeding information to approach their primary care provider or hematologist. Additionally, Dr. James’ initiative like the aforementioned organizations is deft at using social media to increase awareness.

Ladies! Looking for a WWBD clinic near you?

The FWGBD.org site has an excellent locator tool.

Just scroll down on the homepage to find it.

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ISTH Bleeding Assessment Tool

List of Coagulation studies needed for WWBD

To appropriately evaluate the bleeding tendency of a woman or girl at risk for a bleeding disorder, the best first step is to objectively list the bleeding symptoms. One of the best methods to do this is to use the ISTH Bleeding Assessment Tool (ISTH BAT). This is available online via letstalkperiod.ca, academic website (bleedingscore.certe.nl), and the CDC. A typical approach would be if there is an abnormal bleeding score (≥ 6 for females, ≥ 4 for males) a laboratory battery should be considered. This typically includes a CBC, PT and aPTT, Fibrinogen activity, and VWD profile of labs.

The usefulness of the PFA-100 is widely debated and thus not routinely ordered. If this evaluation is negative, then it is reasonable to consider repeating the VWD profile particularly if the results are <100% and ordered with a platelet aggregation study (whole blood impedance or light transmission aggregometry.)

1. Complete blood count: Includes the hemoglobin and platelet count.

2. PT and aPTT: Prothrombin time and Partial thromboplastin time. This “bleeding time” can determine whether there is a significant reduction of certain clotting factors. It is limited in that mild reductions may be missed. FXIII deficiency will not be screened for using PT and aPTT. 3. Fibrinogen: This is a clot-based assay that can be used to screen for fibrinogen deficiency or dysfibrinogenemias. There is also a fibrinogen antigen that can be used if this activity assay is low.

4. Factor assays: Depending on whether there is a family history of a certain factor deficiency, individual factor levels may be sent. This includes FII, FV, FX, FXI, FVIII, FIX, FVII. FXIII is rarely sent due to the prevalence being less than 1 in 1 million.

5. VWD profile: This typically includes the VWF antigen (VWF:Ag) a functional assay (VWF:RCo or VWF:GPIbm) and FVIII (factor 8 activity). Often times this assay may need to be repeated particularly if obtained when severely anemic or during illness. A number of additional studies that can be sent depending on the initial results of the above. Certain labs will use the VWF:CB (collagen binding assay) to screen for type 2 VWD instead of using multimer distribution evaluation (how the VWF looks on a gel and whether all portions of it are present).

6. If the above is negative and there is concern for a qualitative platelet disorder it is reasonable to consider sending platelet studies (typically whole blood impedance or light transmission aggregometry).

Basically, these tests evaluate how well your platelets work and react to certain reagents.

FEATURE

RESOURCES

As part of largely collaborative efforts a significant number of resources for both healthcare providers and patients have been created.

CDC.gov

CDC.gov has a fact sheet about symptoms of bleeding in WWBD

BetterYouKnow.org

(Betteryouknow.org is a collaboration between NHF and the CDC) 1. Postcard advertisements focused in improving awareness of WWBD 2. Menstrual chart and scoring system (Pictorial Bleeding Assessment Chart) 3. Teen booklet focused on discerning between normal and heavy menstrual bleeding 4. Doctor visit preparation list to improve communication between provider and patient 5. Healthcare diary list to focus the conversation on bleeding symptoms and signs 6. Lab test log to compile a list coagulation studies that may have been obtained from various healthcare systems 7. Testing booklet that explains the various coagulation studies important for making an appropriate diagnosis of a bleeding disorder

period.ca: Letstalk

LetsTalkPeriod.ca has an electronic online version of the self-BAT, a self-administered version of the ISTH Bleeding Assessment Tool) assigning a score determining whether a woman or girl is at risk for a bleeding disorder such as von Willebrand Disease

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Hemophilia.org

1. NHF (National Hemophilia Foundation Hemophilia.org) includes MASAC's (Medical and Scientific Advisory Council) guidelines (#251 and 245) focused on perinatal care and general recommendations for WWBD. 2. Victory for women (victoryforwomen.org) website focused on supporting WWBD and creating a community for conversations and general support.

Hemophiliafed.org 1. HFA (Hemophilia Federation of America - HemophiliaFed.org) offers the Women Bleed Too! Toolkit includes HFA/FWGBD brochures focused on bleeding symptoms to help identify women with possible bleeding disorder and educational patient directed materials 2. Sisterhood smartphone app which is an electronic version of the PBAC (Pictorial Bleeding Assessment Chart)

FWGBD.org 1. FWGBD (Foundation for Women & Girls with Blood Disorders - FWGBD.org) has made collaborative efforts with HFA above 2. Library of published papers focused on WWBD and compiled guidelines and an â&#x20AC;&#x153;ask the expertsâ&#x20AC;? option 3. List of clinics and level of resources focused on women and girls with bleeding and blood disorders

Ongoing Research projects in WWBD

There are a number of ongoing clinical initiatives addressing WWBD and I will highlight a few. ATHENA1 is an ATHN DREAM Award project led by Dr. Kristina Haley focusing on characterizing WWBD enrolled in the ATHNdataset. Recently, she described nearly 9,000 WWBD in the largest cohort to date.

Of note, it highlighted our need for better focus on medical management of heavy menstrual bleeding.

The VWDMin (Von Willebrand Disease Minimize Menorrhagia) study (NCT02606045) is evaluating the usefulness of Tranexamic acid and a VWF concentrate in the reduction of heavy menstrual bleeding in adolescent and adult women with low VWF and VWD. It is ongoing and led by Dr. Margaret Ragni from the University of Pittsburgh. The University of Miami Hemophilia Treatment Center has an investigator-initiated surveillance study for women with any bleeding disorder between the ages of 16 – 40. The participants will complete a questionnaire, undergo a physical therapy evaluation, and have on-site ultrasound imaging of their knees, elbows and ankles (the “index joints”) with enrollment occurring at the National LadyBugs Women’s Summit presented by the nSpiration Foundation in collaboration with CHES (Comprehensive Health Education Services.)

Unmet Research Needs

As I see it, there are a number of unmet needs in WWBD. I will highlight a few that I feel need to be addressed soon, and certainly there are more to address.

1. We do not understand how much a congenital bleeding disorder, such as VWD or hemophilia carriage, contribute to the severity or irregularity of heavy menstrual bleeding or any reproductive tract pathology.

To that end we need to focus on the pathophysiology of uterine bleeding (period bleeding and menopausal bleeding). We do not know whether a bleeding disorder leads to anatomic defects or alteration of the blood vessel formation leading to irregular and prolonged periods.

?

2. We do not fully understand why there is a discrepancy in bleeding symptoms and severity of clotting factor deficiency. This is noted particularly in men with mild VWD and in rare bleeding disorders, but it seems to be more pronounced in WWBD. For example, we have not been able to determine why a woman with a FVII level of 35% may bleed more than one with a level of 5%, and why women with hemophilia and mild factor deficiency have similar bleeding tendencies as those with normal levels. Even more of a challenge is in those with low VWF and mild VWD. The range of bleeding is quite large and likely due to the multiple genetic causes of reduction of VWF in humans.

FEATURE

3. We are not able to accurately predict bleeding with day to day events and with procedures, particularly in those with very mild factor deficiencies. Larger prospective studies will be needed to address this issue.

4. The pathognomonic bleeding symptom of hemarthrosis in hemophilia is poorly described in hemophilia carriers and those with mild, moderate and severe deficiency.

Better documentation and evaluation of treatment outcomes will ensure better communication between providers and patients.

onio

. Sid

to Dr

ome My H Records ment Treat

5. We have not been able to prospectively evaluate the role of hemostatic agents and hormonal agents (birth control pills, IUDs, and progestin only pills) in the medical management of heavy menstrual bleeding.

We have not been able to effectively conduct clinical trials in this group due to the inherent challenges of infusions of CFCs (clotting factor concentrates) at the onset of the period. The majority of persons with VWD do not know how to infuse (due to infrequent IV infusions in general), certainly a much lower rate than those with hemophilia. Currently the VWDMin study (NCT02606045) is evaluating the usefulness of Tranexamic acid and a VWF concentrate in the reduction of heavy menstrual bleeding in adolescent and adult women with low VWF and VWD. To date, no prospective study of the role of FVIII or FIX concentrate has been executed in hemophilia carriers, again due to the complexity of infusions on the first day of the period and lack of large enough participation.

6. We need further investigation of the role of imaging, such as musculoskeletal ultrasound and MRI in surveillance of possible joint damage/bleeding particularly in the women and girls with mild and moderate clotting factor deficiency â&#x20AC;&#x201C; more specifically in hemophilia and VWD.

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7. We need a more inclusive registry or surveillance system.

Potentially we could leverage the resources of NHF and ATHN for better longitudinal evaluation and there will be an international effort with ISTH (International Society of Thrombosis and Hemostasis) soon to collect data on hemophilia carriers and VWD patients. Women and girls with bleeding disorders are currently part of the CDC Community Counts initiative (part of the ATHN data collection) and are included in the general authorization into the ATHNdataset. I believe a focused registry is needed to be able to answer some of the questions posed and to encourage larger scale participation.

With continued ongoing collaborative efforts from the patient advocacy groups, medical research societies, government, and pharmaceutical industry we can address the unique medical and research needs of WWBD. In my relatively short career, I have seen a concerted effort and only see continued dedication from the next generation of young investigators.

Dr. Sidonio is an Assistant Professor of Pediatrics, Emory University School of Medicine, Clinical Director of the CHOA Hemophilia Treatment Center, and the Associate Director of Hemostasis and Thrombosis, Department of Pediatrics, Childrenâ&#x20AC;&#x2122;s Healthcare of Atlanta. The focus of his clinical research career has been on understanding the prevalence of congenital bleeding disorders such as low VWF, qualitative platelet disorders or hemophilia carriage in adolescent girls and women with heavy menstrual bleeding and the diagnosis and management of women and girls with bleeding disorders.

Dr. Sidonio has multiple national leadership and/or working group roles in every major hemostasis organization. Notably, Dr. Sidonio is the national PI for a recently awarded grant called ATHN 9 (severe VWD), Mexico Inhibitor Study (natural history of inhibitor development in Mexico) and Emicizumab PUP and Emicizumab ITI study.

FEATURE

Complimentary

&

By: Angela Lambing, MSN, ANP, GNP

Alternative

...Uses in Pain for Bleeding Disorders

P

ersons with bleeding disorders (PWBD) can suffer from many painful experiences, whether it is the result of the needle stick for factor administration or suffering bleeding episodes into a joint or muscle. Additionally, PWBD can suffer traumatic injuries such as sprains or fractures, where pain can result. Given the current climate of the Nation’s Opioid crisis, the ability to receive pain medications via conventional means is becoming increasingly difficult as providers face concerns over censure of prescribing practices, worries of addiction and/or diversion of the medications prescribed to patients, and the potential for patient overdose. As a result, it can be difficult to obtain pain medication when needed. Additionally, it has been well documented that non-steroidal anti-inflammatory pain medications (NSAIDs) can be very helpful for management of pain but are contraindicated in PWBD due to the increased potential for further bleeding as defined by MASAC2.

The United States’ National Center for Complementary and Integrative Health (NCCIH) defines: •

Complementary medicine as a “non-mainstream practice” which can be used together with conventional medicine2.

•

Alternative medicine as “non-mainstream practice” used in place of conventional medicine.

Complimentary and Alternative Medicine (CAM) is further defined by complementary, alternative, and integrative approaches. These approaches are broad-based to health and well-being focusing on treating the whole person including emotions, mental health, social, spiritual, and community.

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There are four distinct domains within CAM including:: 1. Mind-body therapies 2. Manipulative and body-based therapies 3. Natural products 4. Energy therapies

People are now looking at CAM therapy as a potential option to manage pain. Currently, there is limited research in this area with respect to PWBD. This is the first of a 4-part series that will explore the use of CAM with PWBD, reviewing current research studies that explore each of the CAM domains. This article will review the current research available for PWBD within the Mind-body domain of CAM. Below are the different therapies that are considered under the Mind-body domain.

Mind Body Domain of CAM Tai-Chi Yoga Relaxation Hypnosis Magic Glove® Psychotherapy

Biofeedback Cognitive reframing Distraction Buzzy Bee® Neuroplasticity

Tai chi

Psychotherapy

Tai chi has evolved into a graceful form of exercise used for stress reduction and other health conditions. Often described as meditation in motion, tai chi promotes serenity through gentle, flowing movements which allow controlled muscle stretching3. A study was conducted in 2012 with a team of Chinese researchers looking at how Tai Chi might help PWBD’s arthritis symptoms. Results indicated pain, fatigue, and depression decreased while mobility, muscle strength, and quality of life improved with Tai Chi practice.

Psychotherapy, or talk therapy, is a way to help people with a broad variety of mental illnesses and emotional difficulties. Psychotherapy can help eliminate or control troubling symptoms so a person can function better and can increase well-being and healing. Problems helped by psychotherapy include difficulties in coping with daily life; the impact of trauma, medical illness or loss, like the death of a loved one; and specific mental disorders, like depression or anxiety13. In all cases identified within PWBD research, psychotherapy was found to be helpful14,15.

Yoga Yoga involves the practice of specific physical postures, breathing techniques, and sometimes meditation derived from Yoga but often practiced independently to promote physical and emotional well-being5. Behishtipoor and team in 2005 evaluated the effects of yoga on quality of life in children with hemophilia. His findings identified improvement in quality of life, decrease in bleeding episodes and number of school absences, citing that yoga improved the physical psychological, and social aspects of individuals6.

Biofeedback Biofeedback is a mind-body technique that involves using visual or auditory feedback to gain control over involuntary bodily functions including the relaxation of certain muscles, slowing heart rate or respiration, or reducing feelings of pain16. This method has documented positive results17,18,19 although studies were very old.

Relaxation Relaxation exercises and guided imagery has been well documented to assist persons with pain management in a variety of medical conditions. In 1985, a study evaluated relaxation related to frequency of bleeding in hemophilia7.

Although they were not able to prove a decrease in bleeding frequency, relaxation exercises were helpful in the management of pain.

Hypnosis & The Magic Glove® Hypnosis involves progressive relaxation by a trained therapist that allows suggestive behaviors to improve the management of pain and other disorders. Several older references were identified where hypnosis was used in hemophilia in case reports, many where the therapy was used during dental procedures8, 9, 10,11. In each case, the level of distress was reduced as was the amount of factor usage to control bleeding. The Magic Glove® is a hypnotic technique used in children’s centers12. Through focused attention, the child’s imagination changes the sensations, creating decreased sensation or numbness in the area with the “Magic Glove” in place boosting a child’s confidence and comfort in the ability to cope with painful and fearful procedures. There are currently no research studies using this technique in PWBD, although many pediatric hemophilia centers report using this technique successfully.

WHAT’S NEW?

Cognitive Reframing Cognitive reframing is a technique used in therapy to help create a different way of looking at a situation. Therapists often use this technique to help clients look at situations from a different perspective20. This therapy helps people in pain, to view their pain experience in a different way thus allowing them to improve coping of the pain experience.

Elander (2013) studied this technique for males with hemophilia helping them to reduce pain intensity, improve quality of live, reduction of negative thoughts, and active pain coping21.

Distraction & The Buzzy Bee® Distraction as a method of pain management is often used especially in young children. This can be something as easy as watching a movie, playing a video game, or talking with others. Many studies have demonstrated positive results in reducing pain during the distraction

period22,23,24,25,26. Dr. Dunn and her Editor's Note: A comprehensive article team from Nationwide Childrens’ on Dr. Amy Dunn and Hospital in Columbus Ohio her center's practice on recently studied the use of virtual virtual reality can be read reality (VR) during IV insertion in the What's New section with children. This method was of Lifelines for Health's found to be a positive distraction volume 10 (Winter 2017 technique and the desire to VR for Ed.), titled - Pain and the future procedures was positive for Power to Heal along with patients, nurses, and parents27. the Buzzy Bee® This method has also been used in a variety of painful procedures including painful burn procedures in soldiers. The Buzzy Bee® is also a method of distraction using a cold sensation and vibration device to distract children during needle sticks. There are no research studies using this method, although many pediatric hemophilia centers verbalize positive results when in practice.

Neuroplasticity Neuroplasticity is a relatively new process in the community defined as; the ability of the brain to form and reorganize synaptic connections, especially in response to learning or experience or following injury28. Given the newness of this method, there is currently no research in PWBD. The mind-body connection is a powerful relationship. By harnessing the brain and how we view painful experiences can help in improved coping of painful experiences. In many of these methods, the risk of use in PWBD is very low, and should be part of the discussion with the treatment center as additional measures to trial to assist with pain management.

In our next section, manipulative and body-based therapies will be reviewed.

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References 1.

2.

3.

4.

5.

6.

7.

8.

9.

10.

11.

12.

13.

14.

15.

MASAC guidelines for NSAID use; available from: https:// www.hemophilia.org/Researchers-Healthcare-Providers/ Medical-and-Scientific-Advisory-Council-MASAC/MASACRecommendations/MASAC-Recommendations-on-Use-ofCOX-2-Inhibitors-in-Persons-with-Bleeding-Disorders.

National Center for Complementary ad Integrative Health.

Complementary, alternative or integrative health: What's in a name? July 2018. Available from: https://nccih.nih.gov/health/ integrative-health.

What is Tai Chiu; available from: https://www.mayoclinic.org/ healthy-lifestyle/stress-management/in-depth/tai-chi/art20045184 Bak, Won-Sook, Yoo, Myung-Chul, Kang, Hyun-Sook. The Effect of Tai Chi Self Help GroupI Program for Hemophilic Arthritis Patients. Journal Muscle Joint Health. Vol.19 No.1, 71-83, April, 2012.

Definition of yoga. Available from: https://www.merriamwebster.com/dictionary/yoga.

Behistipoor N et, al. The effect of yoga on quality of life in the children and adolescents with haemophilia. International journal of Community based nursing Midwifery. 2015. 3(2): 150-55.

Lichstein KL, Eakin Tl. Progressive versus self-controlled relaxation to reduce spontaneous bleeding in hemophiliacs.

Journal of Behavioral Medicine. 1975. 8(2): 149-62.

Swirsky-Sacchetti T & Margolis CG. The effects of comprehensive self-hypnosis training program on the use of factor VIII in severe hemophilia. Interational Journal of Clinical Exp Hypnosis. 1986, 34(2); 71-83.

Newman M. Hypnotic handling of the chronic bleeder in extraction: A case report. American Journal of Clinical Hypnosis. 1971. 14(2); 126-7.

LaBaw W. The use of hypnosis with hemophilia. Psychiatric Medicine. 1992. 10(4); 89-98.

Federicks LE. The use of hypnosis in hemophilia. American Journal of Clinical Hypnosis. 1967. 10(1).

What is Psychotherapy. Available from: https://www. psychiatry.org/patients-families/psychotherapy.

Magic Glove: Available from: http://pediatric-pain.ca/wpcontent/uploads/2013/04/The_Magic_Glove12.pdf.

Caldwell HS, Leveque KL, Lane DM. Group psychotherapy in the management of hemophilia. Psychological Reports. 1974.

Aug;35(1):339-42.

Mattsson A, & Agle DP. Group therapy with parents of hemophiliacs: Therapeutic process and observations of parental adaptation to chronic illness in children. Journal of the American Academy of Child Psychiatry. (1972). 11(3), 558–571.

16. Cherry K. What is Biofeedback. Available from: https://www. verywellmind.com/what-is-biofeedback-2794875.

17. Varni JW. Behavioral medicine in hemophilia arthritic pain management: Two case studies. Archives Physical Medicine Rehabilitation. 1981. 62(4): 183-7.

18. Lemanek KL, Ranalli MA, Green K, Biega C, & Lupia C. Diseases of the Blood: Sickle Cell Disease and Hemophilia. Handbook of Pediatric Psychology. (2003). p. 321–341. The Guilford Press.

19. Walco GA & Varni JW. Chronic and Recurrent Pain: Hemophilia, Juvenile Arthritis, & Sickle Cell Disease. 1991. Children in Pain.

297-335.

20. Morin A. Using cognitive reframing for mental health.

Available from: https://www.verywellmind.com/reframingdefined-2610419.

21. Elander B, Morris J, Robinson G. Pain coping and acceptance as longitudinal predictors of health-related quality of life among people with haemophilia-related joint pain. European Journal of Pain. 2013. 17(6); 929-38.

22. Elander J, et al. An assessment of the relative influence of pain in coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia.

2009. Pain. 145; 169-75.

23. Elander J ety al. Randomized trial of a DVD intervention to improve readiness to self=manage joint pain. 2011. Pain. 152; 2333-41.

24. Santavirta N. et al. Coping strategies, pain, and disability in patients with hemophilia and related disorders. 2001. Arthritis Rheumatology. 45(1); 48-55.

25. Varni JW. Self-regulation techniques in the management of chronic arthritic pain in hemophilia. 1981. Behavioral Therapy.

26. Dunn A, et al. A feasibility and usability study of a nursing orchestrated, customized 3-dimentional virtual reality environment in children with hemophilia undergoing routine intravenous procedures. 2017. Blood. 130;3691.

27. Buzzy Bee; Available from: https://buzzyhelps.com/.

28. Definition of neuroplasticy; Available from: https://www. google.com/search?rlz=1CAIUCU_enCA882&sxsrf=ALeKk00 nfhh2IoNGhIpUDQvmp3s9wVbV7g%3A1582233639869&ei =J_hOXrO7NIT3-gSax7rQBg&q=neuroplasticity+definition&oq =neuroplasticity&gs_l=psy-ab.1.1.0i273l2j0l8.1020223.1023448. .1024866...1.2..1.356.3898.0j6j8j3......0....1..gws-wiz.....10..0i71j0i6 7i70i249j0i67j0i131j35i362i39j35i39j0i131i67j0i273i70i249j0i20i 263j0i10.W21tlwsvkVw.

Angela Lambing has been a nurse for 40 years; 30 years as a nurse practitioner certified in adult primary care and geriatrics. She spent 15 years as the Henry Ford Hospital HTC nurse, responsible for all aspects of bleeding and thrombotic disorders. She is an expert in pain management in the bleeding disorders community, having completed many research projects in this area. She is widely published and has given talks on a variety of topics, locally, nationally, and internationally. Her last 5 years was spent as a clinical expert and educator for Bayer Health Care. She is now retired, but still focusing her energies and knowledge in the area of bleeding disorders. In the past few years, she has been one of many national experts on pain participating on the MASAC pain task force where she continues to provide support in this area.

WHAT’S NEW?

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YOUR JOINT HEALTH IS IMPORTANT EVERY JOINT BLEED MATTERS 70 to 80% of all bleeds in hemophilia occur in the joints.1 If not treated promptly, even a single bleed can lead to permanent joint or muscle damage.2 This can be true even for people with mild hemophilia.2

IS IT A BLEED?3 During the early stages of a bleed, you may feel a tingling sensation in the joint. It may feel warm, or the skin over the joint may feel warmer than skin nearby.

After a short time, the joint may swell up and become stiff or painful. However, you may not see a bruise with a joint bleed.

What’s important is to go by what you feel, not necessarily what you see.

Blood during a joint bleed, which may lead to permanent damage

SCAN TO TAKE A CLOSER LOOK INSIDE A JOINT

WHAT’S NEW?

PREVENT JOINT DISEASE BEFORE IT HAPPENS The most effective way to take care of your joints and protect them from future damage remains holding to your prescribed treatment routine.4 For decades, prophylaxis with factor has been the most widely used treatment for hemophilia, with a proven track record of reducing bleeds, preserving joint health, and, in some cases, resolving target joints.1,5 Even patients with joint damage can benefit from factor prophylaxis because it can help preserve joint structure and function so that things don’t get worse.4

PROPHYLAXIS with FACTOR is the infusion of factor on a routine basis in order to prevent bleeding.4

TARGET JOINTS are joints with multiple bleeds in a short period of time, leading to chronic inflammation.1,4

A care team member of a hemophilia patient evaluates his knee joint with an ultrasound machine to monitor any changes that may need treatment or attention.

KEEPING FIT SUPPORTS HEALTHY JOINTS Staying active—even if it’s just taking short walks around the neighborhood—is a great way to keep your joints healthy, helping the muscles and tendons that surround them stay strong.3 And consider this: every additional pound you gain puts 4 times the amount of stress on your knees.6 So maintaining a healthy weight with exercise goes a long way toward keeping your joints healthy.

The World Federation of Hemophilia encourages participating in physical activity appropriate to your ability and interest to help keep joints healthy and reduce the risk of bleeds.7 You also can stay proactive by having regular appointments with your care team to monitor the current state of your joint health.

Not all physical activities are appropriate for all individuals. Please consult with your doctor prior to engaging in any physical activity.

References: 1. Quon D. Joint health in hemophilia: latest approaches and advances. Medscape. https://www.medscape.org/viewarticle/908267. Published February 5, 2019. Accessed May 6, 2019. 2. Alavian S, Norton C, Othman S. Living with mild haemophilia and care after a bleed—a qualitative study. J Haem Pract. 2016;3(1):14-23. doi:10.17225/jhp00063. 3. The Hemophilia, von Willebrand Disease & Platelet Disorder Handbook.

Hemophilia of Georgia. http://www.hog.org/handbook/article/3/33/joint-bleeds. Accessed May 10, 2019. 4. Manco-Johnson MJ, Soucie JM, Gill JC; Joint Outcomes Committee of the Universal Data Collection US Hemophilia Treatment Center Network. Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood. 2017;129(17):2368-2374. doi:10.1182/blood-2016-02-683169.

5. Mullins ES, Stasyshyn O, Alvarez-Román MT, et al. Extended half-life PEGylated, full-length recombinant factor VIII for prophylaxis in children with severe hemophilia A. Haemophilia. 2017;23(2):238-246. 6. 51 ways to be good to your joints. Arthritis Today website. http://www.arthritis.org/living-with-arthritis/pain-management/joint-protection/joint-health.php. Accessed May 10, 2019. 7. World Federation of Hemophilia. Fitness and physical activity. WFH Guidelines for the Management of Hemophilia. 2nd ed. 2012. http://www1.wfh.org/publications/files/pdf-1494.pdf. Accessed May 10, 2019. 8. National Hemophilia Foundation. Playing it safe: bleeding disorders, sports, and exercise. HANDI Information Resource Center. 2017. https://www.hemophilia.org/sites/default/files/document/files/Playing-It-Safe_0.pdf. Accessed May 5, 2019. 9. Broderick CR, Herbert RD, Latimer J, et al. Association between physical activity and risk of bleeding in children with hemophilia. JAMA. 2012;308(14):1452-1459. 10. National Hemophilia Foundation. Role of new prolonged half-life clotting factors in hemophilia: new clotting factors for dosing schedule flexibility. HANDI Information Resource Center. 2014. https://www.hemophilia.org/sites/default/files/document/files/PHLBrochure.pdf. Accessed May 10, 2019.

Copyright © 2019 Takeda Pharmaceutical Company Limited. 300 Shire Way, Lexington, MA 02421. 1-800-828-2088. All rights reserved. TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited.

S51791 09/19

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STAY AWARE AND TAKE CONTROL OF YOUR JOINT HEALTH Monitoring your joint health with your care team is a good way to stay aware of any early signs of disease and take the treatment steps necessary to keep your joints healthy long term. Your care team may provide deeper insight from clinical examination, x-rays, ultrasounds, and magnetic resonance imaging (MRIs).1 Nowadays, an ultrasound may be routinely used to identify early changes in the joints, which can help guide treatment. Further, if you have joint pain, an ultrasound can help your care team understand if the pain is from a bleed or due to other causes, such as arthritis.1

It may seem like a challenge now, but your commitment to staying on top of your treatment plan through monitoring with a care team, prophylaxis with factor, and staying active will help keep your joints in step for a healthy future.

REMEMBER FACTOR COVERAGE WHEN YOU’RE ACTIVE Prophylaxis with factor is recommended by the National Hemophilia Foundation (NHF) before beginning your exercise routine.8 Keeping Factor in your body above a meaningful level helps provide coverage to protect your joints.9 Prepare for physical activity by planning your factor treatment schedule beforehand to help lower the risk of a bleed.8,10

Infuse with factor to keep joints in the safe zone

Low Bleeding Risk

100%

Factor levels that are safe for physical activity*

Factor Level (%)

75%

50%

Limited factor levels— use caution with high-risk physical activity

25%

Little protection from bleeds—not safe for strenuous physical activity

0 Infusion

Day 3

Infusion

Day 6

Infusion

Day 9

Infusion

In order to keep your joints as healthy as you can, continue your prescribed prophylaxis routine.

And make sure to talk with your doctor about when to modify your infusions based on your activity level.1

High Bleeding Risk

Day 12

Chart adapted from: Role of New Prolonged Half-Life Clotting Factors in Hemophilia brochure from NHF, 2014. *Not all physical activities are appropriate for all individuals. Please consult with your doctor prior to engaging in any physical activity.

VISIT BLEEDINGDISORDERS.COM FOR MORE INFORMATION ABOUT HEMOPHILIA AND INDIVIDUALIZED FACTOR THERAPY. Commitment to our community is always a priority for Takeda Hematology (previously part of Shire and Baxalta). As a leader in hemophilia research, Takeda continues to innovate on your behalf, developing programs and services that support your efforts each step of the way. Takeda is focused on providing advanced hematology treatments for today and innovating for the future.

WHAT’S NEW?

Are You or Your Child Feeling

MARGIN ! p e l H

Hereâ&#x20AC;&#x2122;s

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by Dr. Gary McClain, PhD

NALIZED? O

ne of the conversations I have most often with my clients is about situations in which they are not being listened to. Ignored. Sidelined.

Treated as if they are invisible.

In a word:

marginalized

They experience marginalization in all areas of their lives, at home, at work, at school. And in the offices of their healthcare providers. Here are a few examples: A client I’ll call Paul is living with a bleeding disorder. He has been doing well with his current treatment approach, he is comfortable with the factor he is using and is confident that he is able to stay on top of his condition. Last week, his doctor told Paul that they want him on the newest sub-Q product and gave him the date on which he will move to this new treatment. Not asked. Told.

Meanwhile, a client I’ll call Natalie recently moved with her partner and children to a new town. And that meant moving her healthcare to a new provider.

Natalie has a bleeding disorder that is relatively rare and awareness among healthcare providers is not widespread. She recently met with a physician she found on the Internet, whose profile included experience in treating hemophilia, so she was hopeful he would be able to treat her. When she told him about her diagnosis, the physician’s tone suddenly changed. He told Natalie that she would need to prove her diagnosis to him, with pictures and imaging. He also told her that with her relatively low factor dosing, she was most likely experiencing a placebo effect. In other words: This is all in your head. Patronized. Disrespected.

FAMILY MATTERS

Mikey is a seventh grader with a bleeding disorder. This is the first year he has been in an all boy’s gym class. His bleeding disorder is well managed, and his parents have worked closely with all of his teachers to make sure they understand his condition and what this means for day-today participation in school activities. His teachers have generally been onboard. That is, all but one. This teacher informed Mikey’s mother that he will not be responsible for any injuries Mikey might receive in class. Last week, the other kids went swimming and Mikey was asked to sit on the sidelines. Marginalized. Humiliated.

Are these examples bringing back any memories or pushing any emotional buttons, based on your own experiences living with a bleeding disorder or as a parent of a child with a bleeding disorder?

And You With a Question: Don’t I/My Child Deserve Better Than This?

A common theme in conversations I have with my clients who are living with chronic conditions is the feeling of being pushed onto the margins of life, or ignored, or disrespected.

And feeling that a condition like a bleeding disorder has automatically branded them in some way. Parents have the same concerns about their children. So, I am going to begin by answering my own question.

Yes, you deserve better. And yes, your child deserves better.

However, as you read in my examples, deserving better sure doesn’t guarantee that’s what you will experience. As you probably already know.

And also, yes, I have some ideas to help.

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You Just Have to Wonder… Where Is This Coming From?

Chalk this up to my mental health professional side, but it is my nature to want to dig in and ask what’s behind someone’s behavior. In this case, the someone who is the cause of you, or your child, feeling pushed to the sidelines. And, to be honest, I also am all about trying to not automatically assume the other person has negative intentions.

Based on my experience, here is what I think causes other people to marginalize an individual dealing with a chronic condition like a bleeding disorder: •

Fear of saying the wrong thing. This can result in ignoring you. Or feeling compelled to say something that is totally not what you need to hear.

•

•

Helplessness. It is human nature to want to help someone in need, and even to feel compelled to step in with a solution when someone else has a need, feeling as if they should know what to do.

Knowing they can’t help you may result in the other person ignoring your needs. Helplessness can also result in denying the existence of your bleeding disorder.

Ignorance. In the lack of knowledge, it is human to make assumptions about conditions like bleeding disorders. And you know how assumptions can leave you or your child feeling.

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Flatfootedness.

Sometimes people mean well but they don’t say it well. Heart is in the right place - foot is firmly in mouth.

Of course, some people are also just plain self-absorbed, if not mean-spirited. However, coping with marginalizing behavior might be less painful if you take the time to think about what might be behind it.

Coping. Starting with Optimism

constantly reminded of: If you see something, say something. So, if someone says or does something that shows lack of understanding, use that moment to gently let them know how you were affected, and what they could do instead. Something like: “When you tell me what I will be doing without talking things over with me, I feel like I am being ignored. Like, what I might want doesn’t matter. So, I’d like to tell you how not to make me feel this way if you’ll be willing to listen.” My clients have reported some positive results with gently speaking up in the moment to be a “patient’ educator – emphasis on patience – when other people marginalize them.

I am going to quote an old saying here: "You can catch a lot more flies with honey than vinegar." So just to follow up on what I said about what might be behind marginalizing behavior by other people, I always encourage my clients to assume goodwill when this occurs and do what they can to adopt a more positive, optimistic approach to these situations.

Here are some ideas: •

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Be compassionate. I am a big believer in compassion, being compassionate toward ourselves, and directing that compassion outward toward others. I think that if you live a compassionate life, you have a firm foundation for weathering the day to day challenges that come your way, and that includes people in your life who just don’t seem to know any better. Letting other people be human helps us not to take everything personally. Compassion is about giving yourself some space and giving space to others.

Look for teachable moments. The New York City subway system has a motto that passengers are

•

Yes, be optimistic. Sure, I am Mr. Optimistic but I am also realistic. Save yourself some heartache by not expecting miracles. Expecting people to think, feel, or behave the way we wish they would, can be a losing proposition. People come around in their own way and in their own time, or, sadly, sometimes not at all. Some of the people you encounter may be willing to look at how they have marginalized you, and even appreciate your feedback. Others not so much. Either way, you tried.

FAMILY MATTERS

Walk Gently but Carry a Big Stick. And Stick Up for Yourself!

I frequently talk with my clients living with bleeding disorders about being empowered. We also talk a lot about empowerment in support groups and meetings. We talk about empowerment because it is so essential to getting what you need as someone living with a bleeding disorder as well as a parent. Empowerment means being an advocate for yourself or your child.

When you find yourself facing off with someone who is marginalizing you, this is a time to engage your advocacy skills. I discussed the gentle approach. Now I’ll address a more direct approach.

Sure, gentle. But firm. If the educational approach doesn’t work, then it’s time to take a more direct approach.

Let the other person know that you won’t put up with the way you are being treated. This might mean saying something like: “I don’t feel like you’re listening to me.”

“We need to talk about what works for me.” “Don’t make assumptions about what I need.” You might also teach your children to advocate for themselves in a more direct manner: “The school nurse has a file about me” or “You can call my parents if you have a question."

Work around the marginalizers. Try to find people who treat you with respect. That might require going over the

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head of a healthcare or education professional, for example, to speak with their boss. Or approaching another staff member who is more enlightened or a better listener. Don’t assume there is only one route to getting what you need.

Have the facts. While that person who is ignoring or otherwise disrespecting you may not respond to your request/demand that they treat you like a real person, they may respond to the facts. So, make sure you’re an expert on your own, or your child’s, bleeding disorder. “Let me show you why I am demanding this.” Information is power.

Use information to regain an equal footing with the person you are trying to communicate with. Nobody can dispute the evidence.

Know your rights. And insist they be honored. For those who ignore you or otherwise treat you like you are invisible - again, gentle but firm. VERY firm, if needed. We have Federal laws that protect individuals living with chronic conditions like bleeding disorders, which include firm guidelines on accommodations, as well as what constitutes an actual Civil Rights violation. Most organizations – workplaces, treatment centers, and schools – have their own guidelines in place. Take the time to get educated on what’s in place for the organizations you or your child are associated with. When you experience marginalization, don’t hesitate to pull out the rulebook.

In the absence of a rulebook, insist on being respected.

Not every situation has a rule or regulation you can quote.

But that doesn’t mean you have to put up with being shoved aside or otherwise overlooked, or that you have to step aside while your child is being mistreated. In the absence of a published guideline, here’s an argument you can stand on: “Just Because. Just because I am a human being like you, and I demand that you show me respect.

That you listen to me. That you take my needs/my child’s needs seriously.” Reserve the right to be a ferocious advocate for your rights and/or your child’s rights.

Get support. It’s so important to have emotional support available when you need it. Taking care of your mental health needs to be a critical component of your overall selfcare plan, as someone living with a bleeding disorder or as

a parent. Build a support network of friends and family who can listen without giving advice, unless you want their advice. Being part of a national organization with local chapters is a great way to get support for you, and for your family, as well as to stay up to the second on the latest treatment developments. Support is power!

Don’t Wait to Deserve. Claim!

But one more time. When someone – a friend, family member, teacher, healthcare professional, and on and on – marginalizes you, they are reducing you to a medical diagnosis. Being marginalized takes away your personhood.

Along with your rights. You also don’t have to sit back and be treated disrespectfully.

If you are living with a bleeding disorder, advocate for yourself. If you are a parent, teach your child to advocate for themselves.

Power up! Seize your power. And hang onto it!

Gary McClain, PhD ,is a therapist, patient advocate, blogger, and author, specializing in helping clients deal with the emotional impact of chronic and life-threatening illnesses, as well as their families and professional caregivers. He works with them to understand and cope with their emotions, to learn about their lifestyle and treatment options, to maintain compliance with medical regimens, to communicate effectively with the medical establishment, and to listen to their own inner voice as they make decisions about the future. His email is: gary@JustGotDiagnosed.com. He welcomes your questions and comments.

FAMILY MATTERS

Going Molecular

by Eric Lowe

The Newest and Coolest BD Treatments

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M a rc h 17, 2020 â&#x20AC;&#x201C; Sigilon Therapeutics, Inc., a biotechnology company has obtained $80.3 million to fund the first-in-human clinical trial (called SIG-001) of their novel encapsulated cell therapy - a process where engineered cells are transplanted into a patient which then produce missing proteins such as insulin, or in this case, clotting factor within the body. The company's therapy platform, called Shielded Living Therapeutics is for hemophilia A and expected to begin in the first half of 2020. The therapy potentially offers convenience, safety, and long-term coverage benefits. With additional investors, total funding for this project reaches $195 million. See more details at: https://sigilon. com/sigilon-therapeutics-announces-80-3million-series-b-financing-to-advanceshielded-living-therapeutics-tothe-clinic/

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April 6, 2020 â&#x20AC;&#x201C; Catalyst Bioscience's sub-q product Marzeptacog alfa (activated), AKA "MarzAA" is entering a phase 3 trial for treating episodic bleeding in individuals with hemophilia A or B with inhibitors, per the FDA's guidance. The trial is called Crimson-1 with planned enrollment for the close of this year, pending any delays of COVID-19. See more details at: http://ir.catalystbiosciences.com/ news-releases/news-release-details/ catalyst-biosciences-announcespivotal-phase-3-studydesign

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here's certainly been some disruption in our lives this year as COVID-19 has taken us on a backseat ride of twists and turns full of confusion, fear, and sorrow. But some things in our life are still in full-swing. Some bad - like the prompt arrival of bills in the mail, tenacious spammers and scammers taking advantage of the moment, and for us young parents - the honorable roles as teachers of our children. And some consistencies are good, such as stability in our medication supply, as well as the progress of newly developing medications. Check them out!

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February 10, 2020 – Novo Nordisk's ESPEROCT® (a recombinant extended half-life factor VIII replacement therapy) is FDA-approved and available in the U.S. for the treatment of adults and children with hemophilia A, non-inhibitors. ESPEROCT® is used to prevent and treat bleeding episodes, as well as control bleeding during surgery. It's intended dosing is every 3-4 days. See more details at: https://www.biospace.com/ article/novo-nordisk-launches-esperoctin-the-u-s-for-the-treatment-ofpeople-with-hemophilia-a/ or at esperoct.com

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March 16, 2020 – Novo Nordisk has paused 3 trials (Explorer 5 - in phase 2 and Explorer 7 and 8 - both in phase 3), which tests the safety and efficacy of prophy treatment of the sub-q Concizumab in a combination of individuals with hemophilia A/B with/without inhibitors. The decision was made by Novo Nordisk after thrombotic events were reported in 3 patients; all survived. An assessment to reinstate or discontinue the trials is in the process. Mads Krogsgaard Thomsen, executive VP and CSO of Novo Nordisk commented, “While it is disappointing to pause the trials, patient safety is of utmost importance to Novo Nordisk...” See more details at: https:// www.novonordisk.com/media/newsdetails.2265611.html

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April 6, 2020 – HEMA Biologics' bypassing agent SEVENFACT® (a coagulation factor VIIa (recombinant)-jncw) is FDAapproved for adults and adolescents (12 and up) with hemophilia A and B with Inhibitors to treat and control bleeding episodes. This is the first new bypassing agent for the treatment and control of bleeding for individuals with inhibitors in over two decades. See more details at: https://ches.education/newsblog/2020/4/6/ hema-biologics-announces-fda-approval-ofsevenfact-for-treatment-and-control-ofbleeding-episodes-occurring-in-adultand-adolescent-hemophilia-aand-b -patients-withinhibitors

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This is by-far not a complete list of newly emerging products within our bleeding disorder community, so we encourage everyone to maintain vigilance and awareness of all news sources to stay up-to-date.

These are exciting times for drug advancement in our community. We cannot disclose now, but at least one of these shows potential promise for our FVIId and GT folks too! Look for an update in our Fall/Winter edition as we revisit their progress.

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April 21, 2020 – Catalyst Bioscience has just successfully completed its phase 2b trial on Dalcinonacog alfa (DalcA), which is a sub-q product to treat individuals with hemophilia B. The prophy goal was met with satisfying FIX activity levels and zero bleeds for all participants. Individuals were dosed daily, but half-life levels show promise for a less frequent dosing regimen. See more details at: http://ir.catalystbiosciences. com/news-releases/news-release d e t a i l s / c a t a l y s t- b i o s c i e n c e s completes -phase -2b -trialsubcutaneous

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Committed to serving the needs of the hemophilia community and physicians

In March 2020, Catalyst Biosciences announced the pivotal Phase 3 study design for MarzAA in individuals with Hemophilia A or B with Inhibitors. The open-label trial will evaluate the efficacy of marzeptacog alfa (MarzAA) a potent, subcutaneously (SQ) administered, next-generation recombinant Factor VIIa variant, to treat episodic bleeding. Catalyst expects that the trial will enroll its first participant before 2020 year end. The Phase 3 Crimson-1 global study will enroll individuals who experience episodic bleeding. Crimson-1 will evaluate the safety and efficacy of SQ MarzAA in the treatment of approximately 230 bleeding episodes in approximately 75 patients, compared with their prior standard of care in a similar number of bleeding episodes.

Catalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia and a research program on subcutaneous (SQ) systemic complement inhibitors. SQ delivery is less invasive, faster to treat, and more convenient than intravenous (IV) drugs currently on the market. Our most advanced asset, MarzAA has completed Phase 2 development in prophylaxis and met its primary endpoint of significantly reducing the annualized bleed rate (ABR) in individuals with hemophilia A or B with inhibitors. SQ dalcinonacog alfa (DalcA) is being developed for the treatment of hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial. We have an early stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B that has demonstrated superiority compared with the Padua variant in preclinical models.

Pipeline Hemostasis SQ Marzeptacog alfa (activated) “MarzAA” Hemophilia & bleeding disorders (rFVIIa) SQ Dalcinonacog alfa “DalcA” Hemophilia B (rFIX) FIX-Gene Therapy Hemophilia B (CB 2679d-GT)

www.catalystbiosciences.com

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Partner

Anxiety is...

Anxiety Normal

by Krystyn Strother

A

nxiety is normal, but we don’t have to let it rule us. We live in a culture that promotes “good vibes only” and a “positive thinking” attitude as a way to be a better person. You see it across social media platforms in many different forms. It’s that general, find the silver lining in all things, kind of attitude that may be adding to your stress and anxiety. One parent I spoke with at a CHES event described to me how the more she tried to think positively, the more anxious she became that she wasn’t doing it right. She felt there was so much pressure to be the positive parent, no drama friend, and never complain about life struggles that instead of boosting her sense of wellness she was increasingly stressed and unhappy.

Feeling anxious is not because there is something wrong with you, it is because there are things right with you. Anxiety, fear, stress…these are

all normal feelings and reactions to what is happening around you that help us navigate life. You won’t be, and can’t be, free from stress and anxiety. Most of the time, it is about recognizing your anxiety, accepting your bad days and the life crises as they arise, and engaging in things that make you feel better (exercise, self-care, mindfulness, meditation) to make it to the other side.

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Being optimistic, practicing gratitude, and looking for the good in a less than desirable situation will absolutely support you in pushing through hard times. There are scientific studies that back this up (Emmons & McCullough, 2003).

However, it does not mean that you won’t feel hurt or disappointed.

Mindfulness and meditation can help us work with those overwhelming feelings but they are not the solution that will solve all of your problems. These

practices are simply tools that work with our central nervous system to facilitate calm, help train our brain to react differently in situations, and provide us moments of rest and ease in an otherwise chaotic world.

Bringing attention/awareness to the physical sensations that come with a difficult experience gives us a chance to learn how we react and practice acceptance. In time, this practice shows us that we can allow unpleasant experiences and still be okay.

Krystyn Strother is the former program director at HUSH Meditation, strategic designer/author of the HUSH meditation curriculum, is a certified meditation instructor, co-founder of NOMAD, “Adventures in Wellness”, and yoga instructor. Krystyn’s yoga classes range from Vinyasa to Yin. In addition to her regularly scheduled classes, Krystyn guest teaches at several yoga teacher training programs throughout the country, speaks at conferences on mindfulness and stress reduction practices, teaches specialized workshops, facilitates yoga + adventure retreats, and conducts continuing education classes for currently registered RYTs. Krystyn holds a certificate of completion in the Yoga of Awareness For Chronic Pain, an evidence-based program sponsored by the Department of Anesthesiology at OHSU. Read more about Krystyn at krystynstrother.com

MIND BODY CONNECTION

Photo courtesy of krystynstrother.com and Stark Photography

Try the following breathing practice next time you feel anxious. Stay with the breath and count.

Notice how you feel before you begin and after.

Recognize your ability to calm and control your breath in a situation where calm and control feel like the furthest thing accessible. And, as always modify as needed to accommodate comfort and accessibility.

Exploring the science behind gene therapy research Gene therapy research has the potential to bring an entirely new option to people with specific genetic conditions.

Many gene therapies are in clinical trials to evaluate the possible risks and benefits for a range of conditions, including hemophilia. HemDifferently is here with gene therapy education, providing accurate information on the basics and beyond.

What questions do you have? Get them answered.

Explore gene therapy at HemDifferently.com.

No gene therapies for hemophilia have been approved for use or determined to be safe or effective.

Š2020 BioMarin Pharmaceutical Inc. All Rights Reserved. MMRC-GTH-0217 3/20

THE 5 STEPS OF INVESTIGATIONAL GENE TRANSFER One method of gene therapy currently being explored in clinical trials is called gene transfer. This approach aims to introduce a working gene into the body to determine if it can produce a needed protein.

STEP 1 CREATING A WORKING GENE A working copy of a mutated gene is created in a laboratory.

STEP 2 BUILDING A THERAPEUTIC VECTOR A therapeutic vector is used to protect the working gene and serves as a transport vehicle for the gene to enter the body. The therapeutic vector is created from a neutralized virus, meaning no viral genes remain inside.

STEP 3 DELIVERING THE WORKING GENE A single, one-time infusion delivers large numbers of therapeutic vectors into the body.

STEP 4 MAKING PROTEINS Once in the body, the new working gene is designed to provide instructions for the body to make the protein it needs on its own. =Proteins.

STEP 5 MONITORING AND MANAGING HEALTH Clinical trial participants are regularly monitored to better understand the safety of the gene transfer and to evaluate its effect on the body.

89 E. Washington Street Hanson, MA 02341-1125

CHES Mission To Inspire awareness and selfreliance for patients with chronic health conditions, their families, and their communities.

Editors in Chief Janet Brewer, M.Ed Eric Lowe

Editor Janet Brewer, M.Ed

Publication Designer Eric Lowe

Contributing Writers Barb Forss Janet Brewer, M.Ed Angela Lambing, MSN, ANP, GNP Eric Lowe Dr. Gary McClain, PhD Dr. Robert Sidonio, Jr., MD, MSc Krystyn Strother

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