Published: Lifelines for Health Spring 2017
It is an incredibly hopeful time in the bleeding disorders community! Longer acting products, gene therapy, and subcutaneous injections all seem to be within our reach. The plethora of new treatments truly boggle the mind. Keeping up with the changes of names for manufactures alone is bewildering! Baxter became Baxalta, which then became Shire. Biogen became Biaverativ, Emergent became Aptevo. While multiple new companies are offering emerging therapies in our community such as Spark, Dimension and uniQure. Deep hope has been ignited again for the first time since recombinant product became available. Treatment promises that may yield fewer infusions per month, subcutaneous injections could make self-infusion and ports a thing of the past. Gene therapy has significantly prolonged FIX activity levels, which substantially increases the length of time one may be infusion-free. Each advancement comes with the opportunity to participate in a clinical trial. This decision however, comes with excitement, responsibility, uncertainty and at times - false hope.
Hemophilia has been in my family for over 7 decades and I have heard “a cure in our lifetime” since 1970. I have personally seen the excitement and uncertainty as we seek new treatment that will make our lives more predictable, better, maybe even.... normal? When carrying my first son in 1988, knowing that I was a carrier, all
I knew was that I was carrying a son and he had a 50/50 chance of having hemophilia. Like every newborn, he was miraculous! A preciously beautiful son who looked normal in every way. I dared to hope that he didn’t have hemophilia. Three days postpartum, cord blood results indicated that he did in fact have severe FVIII deficiency. As a family member of one affected by hemophilia, at least I had some knowledge of what life with a chronic condition might look like. Nothing prepared me for being the mom of a child with a bleeding disorder.
In 1992, I was anticipating the arrival of another beautiful child who would be blessed with hemophilia. Prenatal testing had evolved by that point, that via amniocentesis, we were prepared. Once I learned that he was a boy, I knew in my heart he had it. In truth, I was grateful to have two (2) sons that shared the same disorder. Boundaries and rules would be the same for each. There wouldn’t be one who could pummel the other, while the other one couldn’t pummel them back. My salvation was that I was the one who would infuse them! “Choices have consequences” was our family motto.
In 1991, while pregnant with my second son, I was approached about participating in a clinical trial for recombinant factor. He was the last previously untreated patient (PUP) in the world to be enrolled. Looking back at that time, I realized I knew very little about what adverse events could be anticipated. As a community, we were most worried about HIV and Hepatitis. The scramble to be sure that factor products held no human element of plasma was the goal. My first son was tested for HIV every year until his 6th birthday. The anxiety waiting for those results would reach a crescendo.
With a family member impacted by HIV/Hep C, my first concern was that my sons would also be affected. Signing up for a clinical trial seemed like the best possible choice I could make. Decreased risk of HIV/Hep C was paramount. The trial lasted for five years. It required multiple blood tests at a HTC nearly 2 hours from our home. I look back on that time making those long trips, multiple pokes, the fabulous nurse who bribed him with trucks-one for each hand with a sense of pride that we were doing our part to contribute to science and ground breaking research that would benefit others.
Fast forward to 2002, my second son has developed an inhibitor at the age of 10. THIS was a totally different diagnosis than “plain old hemophilia”. An inhibitor was life changing for our family. Our family was now coping with the challenges of an adverse event. In under 50 years we have seen the development of factor in 1970, (a huge improvement over fresh frozen plasma or cryoprecipitate). Factor then evolved from being plasma derived to recombinant, and then multi-generational factors that eliminated any element of human blood. In that time, our community lost thousands of lives to AIDS and Hepatitis C. Those who survived, now manage as many as three chronic illnesses-hemophilia, HIV and Hep C. Recombinant factor saved my sons from HIV/Hep C, which I will be forever grateful for. Yet, in the last 25+ years, we have seen a 25% or higher rise in inhibitors affecting those with hemophilia A and B from mild to severe. Since 2012, inhibitors are the biggest threat to our community today; finally, surpassing HIV/Hep C.
Now in 2017, there are multiple new treatments in clinical trials that heightens our feelings of hope that finally, THIS might be the one to change our lives. It is both an exciting and perplexing time in the bleeding disorders community. On average it takes 14 years and billions of dollars for a new product to be developed. Choosing to participate in a clinical trial can be a challenging decision. At the 2016 Inhibitor Summits, Dr. Tarantino’s and Dr. Kruse- Jaress’ presented on the “Knowns and Unknowns” of current therapies in clinical trials. There is still so much we do not know when it comes to choosing to participate in a clinical trial. Choices have consequences, with positive and negative effects. If you read the adverse effects of aspirin or acetaminophen, the list is lengthy. When participating in a clinical trial,every symptom is reported. On a trial and have a headache? It will be reported. The headache may have nothing to do with being on a trial, but it could be, so it must be documented. It is probably fair to say that every medication has side effects. This is a big decision, one that may not affect just you. As a parent, you are deciding for your child something that will affect them for the rest of their lives. As an individual, you may have loved ones who could be affected by your decision. Only you can decide what is best for yourself or your family. It is all about making the most informed choice you can possibly make, and your ability to trust in it. So, what can you do as an informed potential participant?
Answer: Arm yourself with as much knowledge as you possibly can.
Clinical trial thoughts to examine:
What phase trial am I participating in?
What is the inclusive/exclusion criteria?
By participating in this trial, does it preclude me from ever participating in another?
Carefully read the Informed Consent Form that contains:
Purpose
Details
Duration
Required procedures such as lab work and how often needed
Key contacts
Is there a patient advisory committee included with this trial and may I participate?
What are the risk/benefits?
Is my current HTC an Investigational Site? If not, is there still a way I could enroll?
Will I be reimbursed for travel if participating in an Investigational Site far from my home?
Will study drugs be included free of charge? For how long? Will my insurance company pick up remaining costs of lab draws, office visits, etc.?
What is the purpose behind wanting to participate in this trial?
Quality of Life?
A feeling of duty/determination to further research for others?
What are your feelings on taking risks?
Avail yourself of resources such as clinicaltrials.gov, PubMed https://www.ncbi.nlm.nih.gov/pubmed/, or Wiley Online Library wiley.com. Trial abstracts are available on those sites and if you would like to read the entire journal articles, ask your HTC provider for them or purchase if available.
13. Read.
14. Talk to friends, family, clergy.
15. Write down your questions before visiting your health care professional. They want you to make a well- informed decision as well.
16. Take your time. There are plenty of opportunities in the pipeline.
17. Trust your gut.
18. Read some more.
19. Be prepared that this may or may not produce the desired affects you were anticipating.