Changing Paradigm of Hemophilia Management: Extended Half-Life Factor Concentrates and Gene Therapy

Seminars in Thrombosis and Hemostasis
Riten KumarManuel D Carcao

Abstract

Management of hemophilia has evolved significantly in the last century-from recognition of the causative mechanism in the 1950s to commercially available clotting factor concentrates in the 1960s. Availability of lyophilized concentrates in the 1970s set the stage for home-based therapy, followed by introduction of virally attenuated plasma-derived, and then recombinant factor concentrates in the 1980s and 1990s, respectively. The subsequent years saw a paradigm shift in treatment goals from on-demand therapy to prophylactic factor replacement starting at an early age, to prevent hemarthrosis becoming the standard of care for patients with severe hemophilia. In the developed world, the increasing use of home-based prophylactic regimens has significantly improved the quality of life, and life expectancy of patients with severe hemophilia. Seminal developments in the past 5 years, including the commercial availability of extended half-life factor concentrates and the publication of successful results of gene therapy for patients with hemophilia B, promise to further revolutionize hemophilia care over the next few decades. In this review, we summarize the evolution of management for hemophilia, with a focus on extended half-life f...Continue Reading

Citations

May 2, 2016·Pathology·Emmanuel J FavaloroJennifer Curnow
Dec 4, 2016·Hematology·Steven W Pipe
Nov 22, 2017·Drugs & Aging·Massimo Franchini, Pier Mannuccio Mannucci
Jul 20, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·Emmanuel J FavaloroGiuseppe Lippi
Apr 19, 2018·Clinical Chemistry and Laboratory Medicine : CCLM·Emmanuel J FavaloroPiet Meijer
Jun 24, 2020·Expert Review of Hematology·Leonard A Valentino, Kate Khair
May 21, 2021·Journal of Thrombosis and Haemostasis : JTH·Olav VerslootUNKNOWN Pharmacokinetic (PK) Expert Working Group of the International Prophylaxis Study Group (IPSG)

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