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Semin Thromb Hemost 2016; 42(01): 018-029
DOI: 10.1055/s-0035-1568877
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

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

Riten Kumar
1   Division of Hematology/Oncology, Department of Pediatrics, The Ohio State University, Nationwide Children's Hospital, Columbus, Ohio
,
Amy Dunn
1   Division of Hematology/Oncology, Department of Pediatrics, The Ohio State University, Nationwide Children's Hospital, Columbus, Ohio
,
Manuel Carcao
2   Division of Haematology/Oncology, Department of Paediatrics, University of Toronto, The Hospital for Sick Children, Toronto, ON, Canada
› Author Affiliations
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Publication History

Publication Date:
15 January 2016 (online)

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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 factor concentrates and gene therapy.

Keywords

hemophilia - prophylaxis - gene therapy
 

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