Recombinant porcine sequence factor VIII (rpFVIII) for acquired ...

Haemophilia (2017), 23, 25–32

DOI: 10.1111/hae.13040

ORIGINAL ARTICLE Clinical haemophilia

Recombinant porcine sequence factor VIII (rpFVIII) for acquired haemophilia A: practical clinical experience of its use in seven patients M . D . T A R A N T I N O , * A . C U K E R , † B . H A R D E S T Y , ‡ J . C . R O B E R T S * and M . S H O L Z B E R G § *Bleeding & Clotting Disorders Institute, Peoria, IL; †Penn Comprehensive Hemophilia and Thrombosis Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA; ‡Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA; and §Department of Medicine, Division of Hematology, Department of Laboratory Medicine and Pathobiology, St Michael’s Hospital, University of Toronto, Toronto, ON, Canada

Introduction: A recombinant porcine factor VIII B-domain-deleted product (rpFVIII; OBIZUR, Baxalta Incorporated, Deerfield, IL 60015, USA) was recently approved for treatment of bleeding episodes in adults with acquired haemophilia A (AHA) in the United States. To date, no clinical experience outside the registration study has been reported. Aim: To describe early clinical experience using rpFVIII for AHA. Methods: A retrospective chart review of seven patients with AHA treated with rpFVIII at four institutions from November 2014 to October 2015. Results: The time to diagnosis of AHA ranged from 5 days to 6 weeks. Six major and one other bleed were treated with rpFVIII following unsatisfactory bypassing agent (BPA) therapy. Good haemostatic efficacy was seen in five of seven cases. rpFVIII loading doses of 100 (n = 6) or 200 U kg 1 (n = 1) increased FVIII activity from