Benefits and limitations of extended plasma half-life factor VIII products in hemophilia A

Abstract

Introduction: Primary prophylaxis with FVIII is the therapeutic regimen of choice in severe hemophilia A; it reduces joint bleeding and associated chronic damage and helps prevent fatal bleeds. However, the high frequency of intravenous injections is a significant challenge for the optimal implementation of prophylaxis, particularly in children and adolescents. Novel therapeutic approaches have been developed to overcome this challenge. FVIII products with an extended plasma half-life can reduce the number of intravenous injections in the frame of prophylaxis regimens and can yield higher trough plasma levels of FVIII. The goal is to avoid all spontaneous bleeding episodes (zero events).Areas covered: This article reviews the benefits offered by the currently licensed extended half-life products and examines limitations, unmet needs, and knowledge gaps.Expert opinion: FVIII replacement remains the standard of care in patients with hemophilia A; however, there have been advances in novel treatment approaches. Non-factor products such as emicizumab offer a promising alternative that warrants more real-life experience and a direct comparison with FVIII replacement.

Keywords: Fc fusion; Hemophilia A; annualized bleeding rate; emicizumab; extended half-life factor VIII; polyethylene glycol; prophylaxis; standard of care.