Recombinant FVIII: the milestone of modern hemophilia treatment

Authors: Mannucci, PM

Affiliations: Fdn IRCCS CaGranda Osped Maggiore Policlin, Angelo Bianchi Bonomi Hemophilia & Thrombosis Ctr, Milan, Italy.

Publication: Haematologica; 2023; 108. 1201–1202

Abstract: On January 19, 1989, the Medical Intelligence section of the New England Journal of Medicine reported the first use of recombinant factor VIII (FVIII) in two patients with severe hemophilia A (HA). This article represents a landmark in its field because it marks two distinct periods of hemophilia care. The first started in the 1970s, when the availability of FVIII concentrates manufactured from human plasma offered the first efficacious form of replacement therapy in bleeding disorders. However, this success story of the 1970s was followed by the mayhem of the 1980s, when many patients with hemophilia developed the acquired immune deficiency syndrome (AIDS) that had been transmitted by concentrates manufactured from pooled human plasma, leading to a dramatic death toll. The scientific research community reacted promptly with the identification of human immunodeficiency virus (HIV) as the cause of AIDS, the development of diagnostic methods, and the demonstration of the efficacy of heating to inactivate HIV in plasma-derived concentrates and so to ensure once again safety.