On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA.
HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out to share with the FDA their hopes, fears, highlights, and trials in treating their conditions. So many great opinions were shared, covering two open-ended topics set by the FDA.
Below is the testimony of Mark Zatyrka, a Blood Brother living with Hemophilia A, Hepatitis C, and HIV. Mark shared with the FDA his concerns with managing the pain cause by his hemophilia treatment while laying out his hopes for the future of treatment and therapy. We hope that after reading Mark’s testimony, you will be encouraged to share your own treatment experiences with the FDA.
My name is Mark Zatyrka and I was born with severe Hemophilia A. I also contracted HIV and hepatitis C in the early 80’s. I’m 34 years old and live in Connecticut with my wife and 18-month-old twin daughters who are also obligate carriers of hemophilia.
Currently I am on an antihemophilic recombinant factor that is plasma and albumin-free. I am currently infusing, intravenously, on a prophylactic schedule (or prophy). So in my case, I infuse every-other day.
My treatment has changed over time. Unfortunately I grew-up infusing “on-demand”. Plus, I often ignored and hid bleeds from my parents. So I would only infuse every couple weeks or even at times once or twice a month. By the time I was in high school both my elbow joints began to lock at about 90 degrees. By the time I was in college, both of my shoulder joints began to lock as well.
It was at that time I switched to prophy. While the prophy didn’t reverse the damage in the joint, I did get a good amount of my range of motion in my shoulders back because when I had all the bleeding I stopped using my shoulders and lost all muscle strength. Infusing regularly enabled me to begin using those joints again, which slowly regained the strength in my muscles around my shoulders and then gave me back much of my range of motion in my shoulders. Unfortunately my elbows were far from repairable.
I have found that switching to prophy has worked well at preventing further damage to my joints.
The only other medications I have taken because of my hemophilia, besides my HIV and hepatitis C meds, has been pain medicine. Everyday I live with severe chronic pain. I have been on oxycotin, oxycodone, celebrex, methadone and morphine.
Pain and pain management has been a huge issue in my life. I’m at a point where I wish I never started on pain medicine. I have been trying to get off all pain meds for years and currently deal with withdrawals almost everyday.
The most significant disadvantages of my current treatment is the fact that I do need to infuse every other day and I still have bleeds, and I still have significant pain. The aspects of my condition that are not improved by my current treatment is that my current treatment does not do anything to reverse the damage that the bleeding has already done.
My number one goal in life right now is to be the best father I can be. Getting repeated bleeds in my elbows and shoulders from carrying them, and knowing I can never play catch with them, makes me incredibly sad.
The treatment that has had the most positive impact on my life has been the fact that my medication is recombinant. Being a recombinant product, I have great confidence in the safety of the medication in terms of viral transmissions. However other risks such as inhibitor development still worry me. I developed an inhibitor when I was younger. We really need to learn a lot more about where inhibitors come from and how to best treat them.
If I could create my ideal treatment it would have zero risk of inhibitor development. After that it depends on how ideal we are talking. A longer acting factor 8 that would allow me to infuse once a month would be great. After that, a subcutaneous shot would be great because of the ease of use and because I’m very worried about the longevity of my veins, as they are in rough shape already. Or maybe an oral medication? Or an implant that creates or releases factor into my bloodstream.
It would also be extremely helpful to have a device, similar to a glucose meter folks with diabetes use, that people with hemophilia could use to check their factor levels. This would lead to much more efficient and cost-effective treatment, in addition to improved outcomes as the information could prevent bleeds.
However, I do want to say that I do not think it’s crazy at this point in time to say my ideal treatment would be to find a cure for hemophilia. I know our federal government had and does provide helpful grants to organizations that help people living with bleeding disorders tackle healthy lifestyles, support treatment centers, and other psycho-social issues, but we really need to invest government dollars towards research to find a cure. I do not consider myself a conspiracy theorist, but I do understand business. Right now, the majority of hemophilia research is being done by for-profit industries who know the value of a life-long patient with a high cost medical condition. It’s a reality. There is no motivation to cure this disorder. That is why I am urging the FDA and our government at large to invest in our community. Invest in the children living with hemophilia. Invest in me, and invest my daughters’ future children. Invest in finding a cure. The science is so close already. We had a major breakthrough in potentially curing hemophilia B recently. This is possible. And if you want to look at what government programs spend on factor reimbursement alone, it makes a lot of sense to invest in a cure now.
If I had the opportunity to consider participating in a clinical trial, I would consider the science behind the drug, how beneficial to our greater community could this drug potentially be, what risks are involved, and where I am with my current treatment. I participated in countless HIV drug studies in the early days of HIV, so I am always open to it if I feel comfortable with the information available.
I would like to thank the FDA for inviting me here today and for your interest in learning more about bleeding disorders. We have made great strides in treating hemophilia, but I honestly believe there is more to do. Thank you again for your time.