This past weekend, theÂ American Society of Hematology (ASH), a worldwide organization of 15,000+ medical and scientific experts on blood diseases,Â held their 57th Annual Meeting to discuss new treatments and research methods.
During this meeting, a group of doctors from around the globe presented the findings of the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET). The SIPPET study showedÂ recombinant factor VIII products were associated with anÂ 87% higher incidence of inhibitor formationÂ than plasma derived factor VIII products among toddler-aged previously untreated patients (PUPs) with severe hemophilia A.
This study was focused specifically on previously untreated toddler patients. The full study is not yet available, but will be published in 2016. It is the first time such a study has been conducted that we are aware of and does raise questions.
HFA encourages youÂ toÂ have an active role in your own health careÂ andÂ toÂ speak with your doctorsÂ regularlyÂ on questions of treatment therapy.
- In the SIPPET study,Â PUPsÂ were patients under the age of six-years old with severe hemophilia A who had never received factor VIII concentrates before. 250+ PUPs from 14 countries and across five continents were studied.
- InhibitorsÂ are antibodies that the immune system develops in response to a clotting factor product used to treat a person with hemophilia.
What HFA Is Doing in Response to SIPPET:
Given the importance of this study, and the implications that it could have for PUPs, HFA is taking the following steps:
- We are consulting with our Medical Advisory Committee to understand more as the study is published.
- We have formally reached out to theÂ Medical and Scientific Advisory Council (MASAC)Â of the National Hemophilia Foundation (NHF) to request that they conduct a full review of this study when it becomes available.
- We have also requested that MASAC consider the temporary suspension of recommendations (or portions thereof) that state any preference for recombinant factor products until the results of the full SIPPET study can be reviewed.
What You Can Do:
While SIPPET focused on previously untreated toddler patients, HFA encouragesÂ all patientsÂ to maintain an active role in their health care.
- Ask Questions:Â Talk to your doctor about this study, your risk for forming an inhibitor, and about the overall care youÂ receive. BecauseÂ every patient with hemophilia is at risk of forming an inhibitor, regardless of therapy product,Â the Centers for Disease Control encourages each patient to be tested annually for one.
- Be Involved:Â Recognize that you play a vital Â role in your health care.Â You have the rightÂ to ask questions, make requests, and learn about all your options.
- Take Action:Â Learn more about this study, and share it with your friends and family.
HFA asks every member of the bleeding disorders community to join us as we advocate every day for safe treatments.Â WeÂ will continue to provideÂ updatesÂ to Â the community as we learn more.Â Together, we are resilient.