Semin Thromb Hemost 2022; 48(08): 904-910
DOI: 10.1055/s-0042-1756188
Review Article

The More Recent History of Hemophilia Treatment

Massimo Franchini
1   Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantova, Italy
,
Pier Mannuccio Mannucci
2   Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico and University of Milan, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
› Author Affiliations

Abstract

The availability first in the 1970s of plasma-derived and then in the 1990s of recombinant clotting factor concentrates represented a milestone in hemophilia care, enabling not only treatment of episodic bleeding events but also implementation of prophylactic regimens. The treatment of hemophilia has recently reached new landmarks. The traditional clotting factor replacement therapy for hemophilia has been substituted over the last 10 years by novel treatments such as bioengineered factor VIII and IX molecules with extended half-life and non-factor treatments including the bispecific antibody emicizumab. This narrative review is dedicated to these newer therapies, which are contributing significantly to improving the long-term management of prophylaxis in hemophilia patients. Another section is focused on the current state of gene therapy, which is a promising definitive cure for severe hemophilia A and B.



Publication History

Article published online:
15 September 2022

© 2022. Thieme. All rights reserved.

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