This past weekend, the聽American Society of Hematology (ASH), a worldwide organization of 15,000+ medical and scientific experts on blood diseases,聽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 showed聽recombinant factor VIII products were associated with an聽87% higher incidence of inhibitor formation聽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 you聽to聽have an active role in your own health care聽and聽to聽speak with your doctors聽regularly聽on questions of treatment therapy.
- In the SIPPET study,聽PUPs聽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.
- Inhibitors聽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 the聽Medical and Scientific Advisory Council (MASAC)聽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:聽
While SIPPET focused on previously untreated toddler patients, HFA encourages聽all patients聽to maintain an active role in their health care.
- Ask Questions:聽Talk to your doctor about this study, your risk for forming an inhibitor, and about the overall care you聽receive. Because聽every patient with hemophilia is at risk of forming an inhibitor, regardless of therapy product,聽the Centers for Disease Control encourages each patient to be tested annually for one.
- Be Involved:聽Recognize that you play a vital 聽role in your health care.聽You have the right聽to ask questions, make requests, and learn about all your options.
- Take Action:聽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.聽We聽will continue to provide聽updates聽to 聽the community as we learn more.聽Together, we are resilient.