Emicizumab for non-severe haemophilia A

Abstract

The treatment and outlook for people with haemophilia has been transformed in the past 50 years through a series of incremental steps that include the introduction of plasma-derived concentrates and their viral inactivation, the introduction of recombinant products of standard and then extended half-life, and the ability of patients to self-infuse at home and use the concentrates prophylactically. With good factor VIII or factor IX (FVIII/FIX) prophylaxis, people with haemophilia have a markedly improved quality of life and a near-normal life expectancy.