Should emicizumab be used in patients with acquired hemophilia A?

Abstract

Emicizumab is currently approved to prevent bleeding in patients with congenital hemophilia A with or without neutralizing antibodies (inhibitors) against factor VIII (FVIII). Here, we present a case-based discussion of its potential use in acquired hemophilia A (AHA), a severe bleeding disorder caused by autoantibodies against FVIII. State-of-the-art management is based on bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) and recombinant porcine FVIII; immunosuppressive therapy (corticosteroids, rituximab, cyclophosphamide) is used to suppress autoantibody formation. Case reports and one series suggest that emicizumab can reduce the risk of bleeding and the requirement for hemostatic therapy until remission of AHA is achieved. Further, it may allow to postpone the start of immunosuppressive therapy or to use less intense regimens. However, the risk-benefit assessment of emicizumab in AHA is difficult because demographic and clinical characteristics are different compared with congenital hemophilia. Prospective clinical trials are needed before the use of emicizumab can be recommended in AHA.