Laboratory Testing for Inhibitors in the Changing Landscape of Hemophilia Treatment Products

Over the last 20 years, technical improvements in the manufacturing process have enabled the development of new recombinant factor VIII and factor IX treatment products used to treat and prevent bleeding episodes in hemophilia patients. These new products are innovative in that they exclude human and animal proteins from preparations, increase the efficiency of recombinant protein production, or extend the product half-life upon infusion. In addition, the FVIII mimetic, emicizumab, became available in 2017 for use in patients who have hemophilia A and inhibitors. Scientific advancements to improve factor replacement therapy have led to considerable quality of life improvements in patients who have hemophilia, but challenges remain.
In particular, the development of neutralizing antibodies, referred to as inhibitors, nullify the therapeutic value of factor replacement in up to 30% and 3% of patients who have severe hemophilia A and hemophilia B, respectively. Testing to detect inhibitors can be technically difficult and, like the treatment products themselves, has evolved in recent decades. The Bethesda assay for measurement of FVIII inhibitors was developed in 1975 and modified in 1995 to the Nijmegen-Bethesda assay, which is the current gold standard method. In addition, techniques to directly measure anti-factor antibodies have been described in recent years. The goal of this webinar is to examine inhibitor testing methods in the context of the changing landscape of hemophilia treatment products.

Видео Laboratory Testing for Inhibitors in the Changing Landscape of Hemophilia Treatment Products канала Hemophilia Federation of America