On May 26 2016, the New England Journal Of Medicine (NEJM) published the results of the Survey of Inhibitors in Plasma Products Exposed Toddler (SIPPET) study. Investigators found that in a randomized study, when previously-untreated patients (PUPs) were treated with recombinant factor VIII, there was an 87% higher incidence of inhibitor development than when treated with plasma-derived factor VIII. Donna DiMichele, MD of the National Institutes of Health (NIH) wrote in an editorial entitled “Hemophilia Therapy — Navigating Speed Bumps on the Innovation Highway” that this new data must be integrated when making product decisions for children with severe hemophilia A.
As the full study data is available for a fee from NEJM, we are working with our team of medical advisors to disseminate the information to our community. We anticipate an updated recommendation from the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) soon. More to come!
- In the SIPPET study, PUPs 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 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 disseminate the information to our community.
- After the release of the abstract in December 2015, we formally reached out to the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) to request that they conduct a full review of this study when it becomes available.
- We have 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 encourages all patients to maintain an active role in their health care.
- Ask Questions: Talk to your doctor about this study, your risk for forming an inhibitor, and about the overall care you receive. Because every patient with hemophilia is at risk of forming an inhibitor, regardless of therapy product, the Centers for Disease Control encourages each patient to be tested annually for one.
- Be Involved: Recognize that you play a vital role in your health care. You have the right to ask questions, make requests, and learn about all your options.
- Take Action: 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. We will continue to provide updates to the community as we learn more. Together, we are resilient.