Prophylaxis for hemophilia A without inhibitors: treatment options and considerations

Abstract

Introduction: Hemophilia A is a bleeding disorder traditionally managed with standard half-life (SHL) factor (F) VIII concentrates. Extended half-life (EHL) FVIII products and emicizumab-kywh, a nonfactor therapy, are newer treatment options. Additional nonfactor agents and gene therapy are expected to reach the market in the near future.

Areas covered: A PubMed (MEDLINE) search from 1962 to April 2020 related to hemophilia A, its management, and the products currently available for prophylaxis was performed to comprehensively review these topics and analyze the benefits and drawbacks of each therapeutic.

Expert opinion: Prophylaxis with SHL FVIII concentrates remains the standard of care for patients with severe hemophilia A and may also be considered for selected individuals with moderate disease. Several years of real-world experience with EHL FVIII, emicizumab-kywh, and other agents in development will be necessary to determine their ultimate roles in the prevention of bleeding and its complications. Gene therapy may not provide a permanent cure for hemophilia A.

Keywords: FVIII replacement; emicizumab-kywh; extended half-life FVIII; gene therapy; hemophilia A; joint bleeding; nonfactor therapies; prophylaxis; standard half-life FVIII.