BloodStream: ‘The Endeavour to Progress’ by Laurence Woollard

    It would be silly of me not to first mention the recent excitement around the Duchess of
Cambridge expecting another baby. Having attended the World Federation of Hemophilia
Congress in Orlando last summer, I know how fond Americans can be of our monarchy. It is
well-known that haemophilia was carried through the royal family. I have to say, without
committing ‘treason’ that Patrick, as a fellow haemophiliac, is every bit as much royalty to
me!

     So, I am honoured to be able to share my perspectives from the ‘across the pond’ for
BloodStream...

     My path into advocacy was cemented with The Haemophilia Society. They are the onlycharity for everyone affected by a bleeding disorder across the whole of the United
Kingdom (UK). With a team of dedicated staff and a network of super motivated volunteers,
they can offer vital help and support where needed to its over 5,500 members and the
wider community.

     In July, the news broke that a public inquiry - a legal investigation conducted by a judge
appointed by the Government - will be held into contaminated blood products. As in
America and many parts of the world, a large proportion of blood products supplied to
patients during the 70s and 80s in the UK were infected with HIV and/or hepatitis C.
This is bittersweet for The Haemophilia Society and activists, who have been campaigning
tirelessly for answers over decades-long. Thousands of people, the majority with
haemophilia and their families, were and still are devastated by this tragedy. Sadly, this
includes my own, with my mum losing both of her parents.

     Consequently, I never got to meet my grandad and often wonder about what his life was
like with haemophilia at my age in 1953. Back then, there was no such thing as preventative
therapy and when he had a bleed, all he could really do was have bed rest and to see it
through. What would he think of my haemophilia treatment and care today?

      As a moderate but with a severe phenotype, I have been able to self-treat prophylactically
since I was around 10-years- old. Just recently, through personalising my care plan with my
specialist clinical team, I have been able to switch to an extended half-life product, which
are now on license here. This has meant that I no longer have to infuse daily, whilst offering
me a certain level of protection.

      However, availability of these and even standard factor concentrates that are safe and
sustainable vary across Europe. Organisations like the European Haemophilia Consortium

      (EHC), based in Brussels – the hub of the European Union - exist to fight the health
inequality amongst approximately 90,000 people 1 diagnosed with a rare bleeding condition
that they represent.

      A snapshot of the disparities in access to treatment and health services across Europe is
illustrated in the EHC’s documentary, Haemophilia Stories 2 . It is a really eye-opening and
emotional account, and validates why having a voice is not a choice to some, but a
necessity. I urge you to watch it.

       In October, the EHC are hosting their annual conference in Vilnus, the capital of Lithuania in
Eastern Europe. This is an important forum for patients, healthcare professionals, the
scientific community and the pharmaceutical industry to share expertise and address unmet
needs. I understand that Lithuania itself, since first hosting the conference in 2009, now
provides prophylactic treatment to children and adults 3 . I have the privilege to be speaking
as part of a company symposium about the patient journey and overcoming the remaining
milestones in haemophilia care from a patient perspective.

      Meetings like that of the EHC do also give a platform to showcase the latest flurry of
research and innovation in haemophilia, with the hope that new therapeutic options could
dramatically change the treatment landscape on a large scale. In fact, it was only last year at
the same conference in Norway that I first heard, ‘cure of haemophilia by gene transfer’
becoming a reality – something certainly my grandad would never have dreamt possible!
The quest to meet patients’ expectations for the future is somewhat evident from the
ongoing clinical trials that I know of in the UK, especially for my friends and peers with
inhibitors. Although I know personally that progress can come at a price, such poignant
losses must not be in vain; we have come this far, let us not stop now!

1 European Haemophilia Consortium. 2013. Mission and Objectives. [ONLINE] Available at:
https://www.ehc.eu/about-ehc/mission- and-objectives/#. [Accessed 11 September 2017]
2 Haemophilia documentary project. 2016. Haemophilia Stories. [ONLINE] Available at:
http://haemophiliastories.eu/. [Accessed 11 September 2017]
3 European Haemophilia Consortium. 2017. EHC Conference. [ONLINE] Available at:
http://www.ehcconference.org/2016-01- 09-15- 17-45/welcome- 5.html. [Accessed 11 September 2017]

Hemophilia- Not Just Physical

This personal essay by Tai-yan Chou-Kudu was featured in Episode 15 of the Bloodstream Pod.

     My name is Tai-yan Chou-Kudu. I am 18 years old and live with severe Hemophilia A. This October, I am starting school to become a Craniosacral practitioner. Craniosacral is a healing modality which allows your nervous system to switch from sympathetic (fight or flight) to parasympathetic (rest and digest). In our modern world, many people are overworked and stressed out, so their body cannot reset in its much needed parasympathetic state. Damage from this stress can store in the tissues of your body for days, months, decades. The body is amazing at healing itself, if it is given the time and space in the right state of parasympathetic relaxation. Craniosacral work is amazing! I have always had a desire to help others heal, and I believe that stems from growing up with Hemophilia. No matter what form it takes, many kids with chronic conditions  have a predisposition for helping others who are experiencing pain and suffering.

   Ever since I was diagnosed at age 3, I've been involved in the Hemophilia community of New York. At 6, I started attending Hemophilia summer camps upstate. These camps were free, and  helped balance out the pain and discomfort of having Hemophilia with the joy of new friendships, and meaningful connections. I remember walking into my cabin that first year, my new home for the week. I was nervous. As I unpacked my bag, I found a post-it note stuck to my bed. It read “Do you want to be my friend?” It was written by another camper who turned out to be the only other girl there with Hemophilia. We became camp buddies and were able to confide in each other and share joyful experiences. That moment, obviously, touched me and has stuck with me -  an early experience of the power of kindness.

   For some reason, the kids at my school were not so open and kind. They would often tease other students who were noticeably different, for example students who were extremely overweight or had learning disabilities. They would frequently make fun of our teachers, even to their faces. Throughout school it was difficult for me to connect to and relate with my classmates. I even went through phases of deep depression. Thankfully, I continued attending Hemophilia summer camp, and came to believe that kids with chronic conditions tended to have a greater maturity and sensitivity. Perhaps coming face to face with our own mortality as well as deeper existential questions triggered this compassion and wisdom within.

   Although the connection between Hemophilia and emotional/mental health is very much unaddressed, I, speaking as a hemophiliac, can tell you that it is not just a physical condition. I believe kids with Hemophilia or any chronic condition are more apt to experience life from a perspective which considers the pain and suffering of others. Therefore, opportunities to take a nurturing role and help relieve the suffering of others are essential. Even for very young kids, there are countless ways to exercise this empathetic nature. Try taking your young hemophiliac to volunteer at an organization called Brown Paper Bag which feeds homeless people in Manhattan and the Bay Area. Or volunteer at an elderly home. I can now clearly see that the compassion and sensitivity we develop early on is a blessing rather than a curse.

  We may struggle because of how different we feel from other kids, but we will often be the ones caring for the downtrodden members of society, understanding the deepest wounds in our friends and family, and, ultimately, displaying one of the most beautiful human qualities:  tenderness in the face of suffering. Hemophilia is a blessing.

To contact Tai-yan, email her at taibubble@gmail.com

Episode 5 - Share Story, Followup - Sleep Resources

On Episode 5, we featured a story on sleep deprivation and the added challenge of getting adequate sleep when also managing either chronic or acute pain. These five resources may provide various tools and tips for how to improve your night's sleep!


Sleep Aids for People with Chronic Pain

http://www.spine-health.com/wellness/sleep/sleep-aids-people-chronic-pain

This article from Spine-Health offers information on sleep aids and techniques that aim to improve quality of sleep for those that suffer from chronic pain. Spine-Health was developed by a group of multi-specialty medical professionals as a resource for people experiencing and seeking advice relating to neck and back problems and related conditions. This article was written by Andrew R. Block, Ph.D., a clinical psychologist at Texas Back Institute, Director of The Well Being Group in Plano, Texas and a Clinical Assistant Professor of Psychiatry at the University of Texas Southwest Medical Center.


Eat Your Way to Better Sleep

http://americannutritionassociation.org/newsletter/eat-your-way-better-sleep

The key to maintaining normal sleep is to maintain a normal eating schedule, and this article explains just how to go about doing so. This piece comes from American Nutrition Association, a not-for-profit, tax-exempt organization made up of healthcare professionals that strive to promote positive nutrition and health.


Better Sleep Found by Exercising on a Regular Basis

https://www.psychologytoday.com/blog/sleep-newzzz/201309/better-sleep-found-exercising-regular-basis-0

Studies show that exercise improves sleep, but don't expect results overnight - it takes time to reap the benefits. This informative article comes from Michael J. Breus, Ph.D., a Clinical Psychologist and a Diplomate of the American Board of Sleep Medicine and Fellow of The American Academy of SleepMedicine. Specializing in sleep disorders, he is one of only 163 psychologists in the world with his credentials and distinction. Dr. Breus appears regularly on Psychology Today, a publication made up of articles about human behavior by renowned psychologists, academics, psychiatrists and writers.


Sleep Quiz

http://www.sleepmanagement.md/Sleep_Quiz.aspx

This sleep quiz is easy and informative. Simply check off symptoms that you experience to find out about your quality of sleep and if you may suffer from a sleep disorder. This quiz comes from the Sleep Management Institute, a board of certified sleep physicians with multiple locations in the United States.


Guided Meditation with Deepak Chopra

http://www.health.com/health/video/0,,20891783,00.html

If you're looking to try out guided sleep meditations, this one, by the renowned Deepak Chopra, is a great place to start. Dr. Deepak Chopra, M.D., is an author, public speaker, and a global leader and pioneer in the mind-body medicine field and New Age Movement. This meditation can be found on Health.com, a site produced by editors and journalists and reviewed by doctors, experts, patients, and advocates. It's articles deliver relevant information by putting health into context in its readers' lives.

Programs, Production, and The Most Important Speech I've Ever Given: A Believe Limited Recap of NHF Annual Meeting & WFH World Congress.

The Anticipation

Believe Limited arrives in Orlando, Florida. As does our equipment. With more to follow.

Believe Limited arrives in Orlando, Florida. As does our equipment. With more to follow.

I attended my first National Hemophilia Foundation (NHF) Annual Meeting in November of 2011 in Chicago (FYI also the 2017 host city). It was an unforgettable experience of community-engagement and education. Social events enabled me, as someone who's primary connection to the community was online, to connect in person for the first time while the symposiums and breakout sessions provided me bleeding disorders education unlike anything I'd experienced before. I knew immediately that Annual Meeting was going to become a staple in my yearly calendar.

During that meeting four-and-a-half years ago, I can also recall discussion of how Annual Meeting was going to be creeping up in the calendar over the ensuing few years in preparation for a landmark gathering in July of 2016: back-to-back NHF's Annual Meeting & World Federation of Hemophilia's (WFH) bi-annual World Congress. Taking place over the course of 10ish days, the combined conferences were anticipated to be the largest gathering ever of people with bleeding disorders.

On one hand, that might sound kinda corny- a giant group of people with bleeding disorders? So what? Why is that important?- but when you consider things like:

  1. On the heels of the blood contamination crisis, we are now living in the era that will see the first generation of people with severe bleeding disorders enjoy an average life-expectancy. 
  2. Potentially breakthrough biotech developments have created a possible future with exciting, radical changes to treatment options for people with hemophilia.
  3. There is booming awareness, activism, and organization for women with bleeding disorders, people with von Willebrand Disease, and people with extremely rare factor deficiencies. 
  4. The 10-fold expansion of WFH's Humanitarian Aid Program (HAP) over the last three years (in 2013 HAP donated approximately 10,000,000 international units (IUs) of factor. In 2016, it's up to 112,000,000 IUs) has enabled rapid expansion of the most robust program in the world serving people with bleeding disorders in developing countries.

Given these considerations and many more, July 2016 had been highlighted in my calendar for some time. Little did I know four-and-a-half years ago, however, that Believe Limited's role during this landmark gathering was going to be a profound one, with numerous programming and video production responsibilities.

The Impact Awards

Teen honorees, Believe Limited hosts, and NYLI guest presenters let loose on stage at the conclusion of The Impact Awards 2016!

Teen honorees, Believe Limited hosts, and NYLI guest presenters let loose on stage at the conclusion of The Impact Awards 2016!

Held following Annual Meeting's Opening Session, the second annual Impact Awards presented by Baxalta (Now A Part Of Shire) and hosted by Believe Limited recognized 43 teens affected by bleeding disorders for their positive impact in one of five categories: Academics, Arts, Health & Wellness, Service & Volunteerism, and Athletics.

Five excellent members of NHF's National Youth Leadership Institute (NYLI) joined co-host Seth Kirschner and I onstage to read the bios of the honorees recognized in each category. Just as I was last year, I was struck by how impressive and inspiring the young people in our community are, and by the enthusiastic support of so many parents, caregivers, and leaders who ensured that these honorees were recognized with gusto!

Our interstitials (the skits and videos between honoree recognitions) were all built around this idea that Seth & I were attempting to raise $109,000,000 to start our own theme park for the bleeding disorders community: Impact World! I'm pleased to say that our wacky idea for infusing (pun alert!) the night with comedy and creating a true awards show environment for our honorees to be empowered by was a success!

The Impact Awards were filmed, so barring any unforeseen challenges, video from the event should be made available in the near future. Congratulations to the 2016 honorees for this well deserved recognition! It was an uplifting way to kick off Annual Meeting!

Powering Through

Participants in Powering Through NHF 2016 (from l-r, Patrick James Lynch, Greta Hayden-Pless, Ali Stroker, and Austin Hanse).

Participants in Powering Through NHF 2016 (from l-r, Patrick James Lynch, Greta Hayden-Pless, Ali Stroker, and Austin Hanse).

Sponsored by National Cornerstone Healthcare Services (NCHS) and produced by Believe, Powering Through is a nationwide, conversational speaker series in which we bring together inspiring individuals from both within the bleeding disorders community and from outside of it to discuss the obstacles and challenges we all face in the pursuit of becoming our best selves.

We've held these events all around the country, but this was our first time at Annual Meeting! NYLI's Greta Hayden-Pless and Gateway Hemophilia Association's Austin Hanse represented the bleeding disorders community, and TV/Broadway actress Ali Stroker- the first performer on Broadway to use a wheelchair- participated as our special guest.

Nine-thirty in the morning a few days into a super busy conference can be a challenging time to get a strong turnout for an event, especially when a trek over-the-river-and-through-the-woods is necessary to find the room! Fortunately, despite these considerations, we had a packed house of energized attendees and the session was superb! Our conversation went in many interesting, inspiring, heartbreaking, and comical directions led by the open sharing of our three excellent panelists. And while there were many in the room attending Powering Through for the first time, there were also a notable number of returning attendees- always a great sign for a program.

I continue to take so much pride in producing these conversations for our community, and I'm thankful that we have recorded every single one so that whether you are in the room or not, everyone has the opportunity to engage the material. Check out video from previous conversations (and soon, from this one as well) at PoweringThrough.org. A huge thank you to NCHS for continuing to be a supportive, thoughtful partner in the ongoing development of this program, and to NHF for including Powering Through in Annual Meeting's agenda this year!

Video Production

Believe Limited: A Dream Team (from l-r, Joshua Sterling Bragg, Lauren Feighan, Andrew Gaul, Rob Bradford, & Brad Reeb).

Believe Limited: A Dream Team (from l-r, Joshua Sterling Bragg, Lauren Feighan, Andrew Gaul, Rob Bradford, & Brad Reeb).

Believe always films the sessions that we host, so seeing us walking around with cameras and sound equipment is nothing new, but this year our video production responsibilities expanded as we produced content for Annual Meeting's Opening Session and Awards Ceremony and were hired by WFH to be the official videographer of World Congress! 

Interviewing Langston Bias, son of NHF CEO Val Bias, during the Opening Reception for WFH's World Congress. Photo courtesy of Robin Bias.

Interviewing Langston Bias, son of NHF CEO Val Bias, during the Opening Reception for WFH's World Congress. Photo courtesy of Robin Bias.

In practice, this meant that throughout both conferences, we had 1-2 (and on occasion, 3) production teams spread out to record various sessions, interviews, and events. Additionally, I'm quite proud to point out that video Believe produced in Senegal last fall for WFH's HAP was also shown in front of an audience of thousands during the Opening Session to World Congress.

How all this content is going to be packaged and distributed remains to be seen (though some has already been released through WFH's social channels). Keep an eye on WFH's & Believe's social channels for updates.

My Most Important Speech To Date.

Photo from my plenary speech on July 27th [courtesy of WFH's The Congress Daily].

Photo from my plenary speech on July 27th [courtesy of WFH's The Congress Daily].

Several months back, WFH asked if I would be interested in being one of the plenary speakers at World Congress. Given my experience with public speaking, my passion for WFH's work, and the incredible opportunity to speak to international leaders and activists in our community, I didn't give it a moment's thought before saying, "Yes absolutely I would love to thank you!"

Photo of plenary, courtesy of NHF Board Chair Jorge de la Riva.

Photo of plenary, courtesy of NHF Board Chair Jorge de la Riva.

To be honest, though I think about this community and my mission constantly, I didn't give the construction of this particular speech much thought until World Congress actually began. And as days went by and other responsibilities were checked off the list, I started to dig deeper on exactly what point I wanted to make, and how I wanted to make it. I kept coming back to this driving thought: 

In a room full of hundreds of doctors, pharmacists, nurses, psychologists, foundation directors, and other impressive community members from all corners of the Earth, what do I have to offer that is uniquely valuable and therefore worthy of this incredible audience's time during this precious, rare gathering?

I'm not a big "notes guy," so most of my speeches are in a very minimal outline form, though given the importance of this particular opportunity, I scripted my closing remarks precisely. WFH does have video from this that I look forward to sharing when it's released, but in the meantime, here is a selection from my closing remarks that speak to the heart of how I now think about self-care:

My journey and understanding of self-care, like many of yours I’m sure, has evolved. I no longer consider it simply self-infusing, stretching and strengthening, and the maintaining a well-rounded diet. Nor is it enough to understand trough levels, insurance policies, and legal rights. For me, self-care is now the unique application of my gifts and skills for the betterment of this community. I am compelled to do the work I do, in the memory of my brother, in order to make sense of losing him. In order to manage the pain of losing him. In order to understand my place in the world. More than utility, that is empowerment, and that is now my baseline for self-care.

I have every intention of continuing my self-care practice that now includes creating meaningful content and experiences for and about people with bleeding disorders. But I acknowledge that I have the luxury of a privileged definition of self-care, a luxury not shared by approximately 75% of hemophiliacs around the world.

The privileged amongst us, most of whom enjoy this privilege by the random fortune of birth location, we belong to healthcare systems from which we benefit greatly. Yes, there are challenges, but on the whole and by comparison, we are very fortunate. This privilege, in my opinion, comes with a responsibility to participate meaningfully in this system. To my fellow patients from the developed world, and particularly those from the United States, I implore you to speak to the reps from the pharmaceutical manufacturer who’s product you use- or go online and educate yourself- learn about the Corporate Social Responsibility of your company. Learn what their position and activity in the developing world is, and urge them to do more. Speak to leadership at your local hemophilia patient organization about potentially twinning with an emerging national member organization. Speak to philanthropists or people in positions of great influence about your condition, and your international community. Encourage financial donations so that WFH can afford the increased shipping costs, training costs, and diagnostic costs that are vital to creating sustainable and predictable care for our international brothers and sisters.

I am young, but I am neither naive nor uniformed. This is hard work. It will take a long time. But in this room, this very room right now, sits leadership with unique expertise and capabilities from all around the world. Realizing a vision of treatment for all necessitates international leadership in the bleeding disorders community prioritizes this vision, and refuses to allow our privilege, local challenges, or other factors to derail that focus. 

Our global community is dependent upon us. Let’s continue to ramp up the efforts for our brothers and sisters who are most in need so that one day, they too may enjoy the luxury of a privileged definition of self-care. Thank you.

The next day, the speech was featured on the front page of WFH's The Congress Daily. Click this photo to read The Congress Daily's recap.

The next day, the speech was featured on the front page of WFH's The Congress Daily. Click this photo to read The Congress Daily's recap.

I've always found it effective to speak from the courage of my convictions with both precise articulation and real human emotion, and this experience was no different as evidenced by the couple dozen people who lined up afterwards to speak with me about the address. People from Egypt, Australia, Korea, Brazil, France, Denmark, and on and on. I didn't anticipate this. I stayed present and attentive to each person who approached, but when that ended and I had a moment to reflect, I was floored. How, on Earth, did I get here, did Believe get here? And, more importantly, with this level of interest in my mission, what do we do next?

Following the morning plenary speech, I hosted a Q&A on my remarks and the power of content in the Exhibition Hall's Showcase Theatre.

Following the morning plenary speech, I hosted a Q&A on my remarks and the power of content in the Exhibition Hall's Showcase Theatre.

At a Question & Answer I hosted later that afternoon, there was an opportunity to engage a bit deeper on the topic of how content specifically has the power to influence hearts and minds, and how I believe we can increase the use of content for these purposes with regards to improving the overall quality of life for the international bleeding disorders community. I'm excited to say I had some intriguing conversations with many likeminded people and look forward to where conversations about this topic go from here.

So what do we do next? Where do conversations go from here?

In addition to what I've recapped above, the joint conferences also consisted of numerous coffeeshop conversations, dinner meetings, and spontaneous spitballing sessions, as well as my attendance at numerous breakout sessions on a wide range of topics, all of which rounded out an incredibly powerful and momentous two-week experience in Orlando.

Spending time during WFH's Farewell Dinner with some powerful advocates (from l-r Patrick James Lynch, Jenna Lovell, Joelle Palmatier, Michelle Cecil, Chloe Christos, and Jorge de la Riva; photo courtesy of Michelle Cecil).

Spending time during WFH's Farewell Dinner with some powerful advocates (from l-r Patrick James Lynch, Jenna Lovell, Joelle Palmatier, Michelle Cecil, Chloe Christos, and Jorge de la Riva; photo courtesy of Michelle Cecil).

It's tempting to bask in the energy and stimulation of all this and feel accomplished, and on one hand, that's completely fair- Believe Limited did an incredible amount of work in preparation for and during these two meetings, and the feedback from our efforts has been strong; credit where credit is due!

On the other hand, those living in the developing world don't benefit from our sitting back and relishing our accomplishments. Women with bleeding disorders can't make strides in their awareness and advocacy efforts if activists and allies feel as though the work is over and grow complacent. And patients within the plentiful US need guidance to help navigate a rapidly evolving product market within a complex healthcare system.

The combined Annual Meeting & World Congress extravaganza of 2016 has been anticipated by many of us for years, and now, it's over. Let's use some down time this August to reflect and recharge, then let's make sure that the energy, connections, and momentum created this summer carries into our work this fall. Let's use the inspiration of such incredible meetings to motivate us to work harder and with even greater focus on addressing unmet needs and improving care. Treatment For All is possible, in ever sense of the phrase if we continue to work hard and work together on making that aspiration a reality.

About the Author

Patrick James Lynch is the CEO and Co-Founder of the award winning digital content agency Believe Limited. He is the 2013 Recipient of HFA’s Terry Lamb Award & the 2014 Recipient of NHF’s Loras Goedken Award. Patrick lives with severe hemophilia A dedicates his work to the memory of his little brother Adam. @pjlynch.

European Medicines Agency Reviews Inhibitor Development Risk in Hemophilia A Treatment

In response to the SIPPET study that was published this spring by the New England Journal of Medicine, the European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing products and the risk of inhibitor development for initial hemophilia A treatment.  This is not the first time the that this body has considered the risk of inhibitor development with recombinant factor VIII products; it also did so in 2013 and 2014 in response to three separate studies related to risks associated with recombinant factor VIII products.  Following its review PRAC will make a set of recommendations, after which the Co-ordination Group for Mutual Recognition and Decentralised Procedures-Human will adopt a position.  Source: "EMA Starts Another Review of Factor VIII-Containing Products" via Medscape.com

 

 

Blood Donations Tested for the Zika Virus in South Texas

Individuals donating blood through the South Texas Blood & Tissue Center (STBTC) are being asked to sign a voluntary consent form for testing of the Zika virus.  While this testing involves no change to the donation process itself, the STBTC views it as a precautionary measure to ensure the utmost safety of the blood supply and hopes for full participation by donors.  Given the continuing emergence of information on the Zika virus and the fact that those affected do not always display symptoms, this testing enables detection of infection that might otherwise go unnoticed.  "Our preference, as health care providers, is to have all donations tested for Zika,” Dr. Rachel Beddard, Medical Director of BioBridge Global, states.  The STBTC is one of, "a handful" of blood centers currently offering Zika virus testing.  Visit the South Texas Blood & Tissue Center for full information

As Restrictions on Hepatitis C Drugs Lift, Issues on Covering Cost and Increasing Accessibility Remain

As Medicaid, Private Insurers Begin To Lift Curbs On Pricey Hepatitis C Drugs more patients are gaining access to medications with a cure rate of over 90%. The increased access however has not been without significant legal and lobbying efforts as questions over lifting accessibility restrictions and covering the hefty price tags of these drugs are grappled with by states, government agencies, drug manufactures and commercial insurers. While an average 12-week course of treatment can currently range between $54k and $95k, this price is expected to decrease as more drug treatments come on the market and competition increases. Executive director of the National Association of Medicaid Directors conveys that the desire to make these drugs available to everyone in need is there however, "with the prices they're commanding, something has to give."

Community Writings on the SIPPET Study

Community education and dialogue play an important role in shaping how the bleeding disorders community processes, understands and applies scientific research. This is certainly true when it comes to the recently published results of the SIPPET (Survey of Inhibitors in Plasma-Products-Exposed Toddlers) study and its implications for treatment options. Following circulation of BloodStream host, Patrick James Lynch's article, The SIPPET Study & MASAC’s Recommendation: What Does It Means For Me?, we learned of additional writings by community members aimed at helping to deconstruct the specifics of the research and translate the scientific findings into important considerations for individuals and families.  

Trained immunologist and community member Murali Pazhayannur has created a site, The SIPPET Studied which helps to simplify the study and its findings, while also posing thoughts and questions about MASAC's recommendations. Additionally, community member Michael Birmingham's What the heck is a SIPPET, and why should I care? post lends his voice to break down what exactly was examined in the SIPPET study and how its findings might inform decisions about treatment. 

Follow BloodStream on Facebook and Twitter for additional SIPPET related information and discussion.

 

The SIPPET Study & MASAC’s Recommendation: What Does It Means For Me?

Disclaimer

I am not a doctor or a medical professional. I am, among other things, a writer with severe hemophilia A and a tolerized inhibitor. Following the article I wrote in January summarizing the SIPPET abstract, the National Hemophilia Foundation (NHF) engaged me to write a patient-facing article summarizing NHF’s Medical and Scientific Advisory Council’s (MASAC) recommendation in response to SIPPET’s publication. 

In agreeing to do so, my goal was to help patients and families understand MASAC’s recommendations related to the SIPPET study on inhibitor formation, and to empower their decision making in choices about care.

To improve my understanding of the issues at hand, I was invited to be a guest listener on MASAC’s conference calls discussing SIPPET and was copied on the ensuing emails with draft after draft of what is now a completed recommendation. I hope this article helps provide some clarity on what is admittedly a complicated set of study results. To that end, the article is written in my language, not in dense medical-ese. 

Once again, I am not a medical professional, and any decision about you or your child’s medical care should be made by you, your family, and your child in conversation with your usual healthcare team. 

Opening

One of the many challenges that come with a hemophilia diagnosis is that you sorta kinda have to become something of a medical expert. And an insurance expert. And a packing expert (factor can fill a suitcase in no time!). 

Read More

Emicizumab (ACE910) Shows Promising Results for Treatment of Hemophilia A

Promising results have been reported in early clinical trials of Emicizumab, otherwise known as ACE910. This subcutaneous therapy is being developed for individuals with hemophilia A, with and without inhibitors. ACE910 has been on a fast-track approval process since September 2015 when it was given breakthrough therapy status by the U.S. Food and Drug Administration. Subsequent study is currently being conducted by the makers of the drug, Tokyo-based Chugai Pharmaceutical Co. Ltd. Its hope is that ACE910 will soon offer a new prophylactic treatment option to individuals with hemophilia A.

 

Shire/Baxalta Merger

On June 3, 2016, Shire announced its combination with Baxalta in a $32 million merger that will notably expand its global reach. The combination positions Shire as the, “global leader in rare diseases.”  In addition to the significant anticipated revenue, the announcement also signals substantial development and innovation of next generation treatment therapies.  Read more about the combination here.

Recombinant Treatment for Hemophilia A Receives FDA Approval

Long-lasting recombinant treatment for hemophilia A, AFSTYLA, has received FDA approval. The new drug, which its manufacturer CSL Behring announced as available today, “offers patients an opportunity for excellent efficacy with a strong safety profile and twice-weekly dosing--potentially helping patients to fit treatment into their active lives,” says Lisa Boggio, Assistant Professor of Internal Medicine, Hematology and Oncology, Clinical Director of the Rush Hemophilia and Thrombophilia Center, and AFFINITY clinical development program study investigator. Read more about AFSTYLA and its approval here.

 

Spark Therapeutics Reports Promising Gene Therapy Results

After decades of study, gene therapy is showing exciting possibility for the treatment of hemophilia B patients. Researchers at Spark Therapeutics have reported promising results in a study of four hemophilia B patients who were injected with viruses containing the gene that codes for factor IX. Factor IX protein which clots blood is otherwise absent in the bodies of hemophilia B patients. While the small study size is keeping scientists from declaring an overall cure, the consistently positive results observed in each of these four patients is being received by scientists as quite encouraging for future one-time treatment possibilities for hemophilia B. In addition to the optimism regarding a potential cure for segments of the hemophilia population, these findings also have encouraging implications for the treatment of several other rare diseases.

Precision BioLogic Donates Coagulation Diagnostic Products Through the WFH Humanitarian Aid Program

Last week Precision BioLogic announced its donation of coagulation diagnostic products through the World Federation of Hemophilia (WFH) Humanitarian Aid Program. The donation delivered products to the University Teaching Hospital in Yanoudé, Cameroon to aid in the diagnosis and treatment of individuals with bleeding disorders. Executive Vice President of Precision BioLogic, Paul Empey says, “We’re excited to make this donation and directly support WFH’s efforts to close the gap in care between those with bleeding disorders who receive proper diagnosis and treatment and those who do not.” The WFH Humanitarian Aid Program seeks to ensure treatment for all patients with bleeding disorders despite current limitations in access to care in developing countries throughout the world. Learn more about the WFH Humanitarian Aid Program here.

Let's Talk Period

Queens University researcher, Dr. Paula James, has developed a self-administered bleeding assessment tool (self-BAT) to aid women in determining whether they may have a bleeding disorder. Bleeding disorders in women frequently go undiagnosed as individuals are often unfamiliar with the symptoms and/or mistake them for normal. The website LetsTalkPeriod.ca which houses self-BAT, “aims to increase awareness of the signs and symptoms of bleeding disorders.” Through increased understanding of what constitutes abnormal bleeding, women who suffer from bleeding disorders can better access treatment to limit unnecessary health complications.