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Profile of efraloctocog alfa and its potential in the treatment of hemophilia A

Authors George L, Camire R

Received 24 February 2015

Accepted for publication 30 March 2015

Published 24 April 2015 Volume 2015:6 Pages 131—141

DOI https://doi.org/10.2147/JBM.S54632

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Dr Martin H. Bluth

Lindsey A George,1,2 Rodney M Camire1–3

1Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; 2Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; 3Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

Abstract: Hemophilia care has improved dramatically over the past 50 years, evolving from plasma concentrates, to purified plasma proteins, to recombinant clotting factors. These collective developments allowed for home delivery of on-demand and prophylactic treatment, resulting in the reduction of hemophilia morbidity and mortality and improved quality of life. Although efficacious in treating bleeding, conventional factor products' half-lives require frequent venipuncture, which remains a significant burden to patients. Despite the remarkable advances in hemophilia care, no improvements have, until now, been made to the pharmacokinetic properties of factor products. Multiple strategies have more recently been employed to generate novel bioengineered products that, with great hope, represent the next wave of progress in hemophilia care. The use of these products will undoubtedly raise important discussion about choosing conventional factor over new long-acting factor products. Incorporation of these therapies into clinical care is accompanied by unanswered safety questions that will likely be evaluated only in postmarketing surveillance analysis. Further, these products may change current treatment paradigms with unclear cost repercussions and feasibility. This paper will review efraloctocog alfa (FVIII-Fc) and its role in the treatment of hemophilia A.

Keywords: hemophilia A, factor VIII, Fc fusion, bioengineered products, efraloctocog alfa

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