MASAC Document #243
The following recommendation was approved by the Medical and Scientific Advisory Council
(MASAC) on June 17, 2016, and adopted by the NHF Board of Directors on June 28, 2016.
The development of inhibitors (neutralizing antibodies to factor VIII) is the most
significant current treatment-associated complication of hemophilia A, affecting up to 30%
of previously untreated patients (PUPs) treated with factor VIII (FVIII). (1) Inhibitors to
FVIII can occur with both plasma-derived and recombinant FVIII concentrates. The rate
of inhibitors observed in PUPs is unacceptably high, therefore clinical trials in this patient
population are critically important to efforts to reduce inhibitor formation.
Results were recently published from SIPPET, a study which analyzed data from 251 PUPs with
severe hemophilia A who might have received transfusions of blood or blood product…