Coalition for Hemophilia B 8th Annual Symposium

Today I attended my first Coalition for Hemophilia B Annual Symposium (the group’s 8th annual) in New York City. What a terrific event. I got to meet several of our Rare Patient Voice members and I learned a lot.

I’d like to share some of what I learned today. Since some of it applies equally to Hemophilia A and other bleeding disorders, I’m sending this beyond our Hemophilia B members.

Dr. Christopher Walsh of Mt. Sinai Hospital in New York spoke on what’s new in Hemophilia. This was incredibly interesting, as not only was he able to draw attention to significant happenings (and it is good to say there are several), but he was able to say it in a very clear, understandable way. Key points:

  • Hemophilia is steadily heading toward a cure.
  • Hepatitis C is (or will be) history. The new drugs can treat it in almost all patients. Many are and will be oral pills, much better than the past series of injections. So if you have hepatitis, there is no reason now not to be treated.
  • Bleeds are preventable! You just need to get your factor. He believes in higher levels of factor, there are no toxicities. Even one bleed can destroy joints. You shouldn’t do on demand treatment any more.
  • Six companies are coming out with long acting factor (Pfizer, Baxter, BiogenIdec, CSL Behring, Novo Nordisk, and Emergent). They could be used with the current frequency to reduce bleeds even more than current products. You can’t overdose on these factors. So now there are no excuses for preventing bleeds.
  • Even mild patients can bleed and he has seen joint problems with milds. So milds should be taking factor also.
  • “The mindset of bleeding should be thrown away. No excuses.”
  • Gene therapy – the six patients who had this trial are all still OK, several years later. At high levels there could be an immune response that could cause liver damage. The Asklepios Factor IX give a 10 times greater response than normal factor.
  • The new CRISPR (clustered regularly individually spaced short palindrome repeats) method of gene therapy is marvelous and may be 15-20 years away from usage.
  • Cell therapy and Gene therapy will converge in the future to correct the gene “mistake” that causes hemophilia; there is nothing stopping this, it is inevitable.
  • To summarize, we now have protein therapy, we are starting gene therapy, and in the distant future will have cell therapy.
  • Inhibitors – he hasn’t seen any news this year; Hemophilia B seems to have fewer inhibitors, but those that have them are more difficult to treat; He doesn’t see any difference with prophylaxis or switching of brands. But he doesn’t see new products coming out that specifically reduce inhibitors.

Carla from CSL Behring spoke in insurance issues.

  • The NHF has a Personal Health Experience Stat Sheet available on the NHF website. This is a great tool to figure out your needs. Do it before you need it. Can do online or print out and do paper and pencil. It is not intimidating.
  • There has been a shift to consumer focus in the market place, which places more responsibility on you.

Wendy Kaufman conducted an interactive session on Positive Assertiveness.

  • She explained the AEIOU steps for being assertive
    • A – Acknowledge the other person’s positive intention
    • E- Express your thoughts and feelings
    • I – Identify your proposal or plan
    • O- Outline the outcome you anticipate and its benefits
    • U – Understand each other? Check to make sure

Val Bias, CEO of the NHF, spoke on genotyping.

  • The NHF offers free genotyping, which increases our understanding and will lead to scientific breakthroughs
  • This is happening for Hemophilia A and B patients and carriers, and they are talking about it for Von Willebrands.
  • No one will own the data – the four partners doing this will not own your data
  • They are waiting to get 5,000 samples

Linda Pollhammer, and Nurse Educator, spoke on overcoming challenges.

  • She emphasized the importance of logging infusions (on paper or via app)
  • Call the HTC for any head injury, even if minor. One patient had what seemed to be a minor event where a branch hit his head while he was mowing the lawn, and he actually died the next day in the middle of making a presentation.
  • In your HTC visits tell them about all medications you take, even over the counter vitamins and herbal supplements, as they can interact.
  • Exercise – stronger muscles protect joints

Pete Dyson, a Hemophilia B patient and now a middle school teacher and coach spoke.

  • His grandfather had Hemophilia B, died recently at age 90, went through the days before factor. He was an inspiration and mentor to Pete.
  • He played college basketball and baseball
  • He encourages all Hemophilia kids to get involved physically

About the author: Wes Michael, President and Founder of Rare Patient Voice, has been involved in rare and orphan diseases since 1998, interviewing and surveying patients, caregivers, physicians, nurses and advocacy leaders. Wes has more than 30 years experience in marketing research, and more than 15 years in healthcare marketing research.

{ 2 comments… add one }
  • Lynn Sanders December 27, 2014, 8:32 pm

    Hi Wes,
    MY name is Lynn Sanders and I’m the Founder/President of the Ehlers-Danlos Syndrome Network CARES foundation. I started this EDS foundation 9 years. We have started 3 EDS research projects. We have a proposal already for our 4th EDS research project, we just need more money to start this research.
    We also do awareness, advocacy, support etc…We are interested in your group.
    My phone is 262-514-2851 or you can email me. Thanks so much, Lynn Sanders

    • Ivy May 6, 2015, 12:26 pm

      Will there be a gene therapy or treatment for eds in the future? Decades?

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