title: Recombinant porcine sequence factor VIII (rpFVIII) for acquired haemophilia A: practical clinical experience of its use in seven patients.
abstract: BACKGROUND
  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.
  
  
  OBJECTIVE
  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 <1 to 9% at baseline to 109-650% within 0.25-7 h in six of seven cases. Subsequent median doses ranged from 30 to 100 U kg-1for 3-26 days. No rpFVIII-related adverse events were reported. Three patients survived with inhibitor eradication, one with persistent inhibitor, two died with inhibitors present and one was discharged and later died from unrelated causes.
  
  
  CONCLUSIONS
  rpFVIII showed good haemostatic efficacy with no recurrences in most cases, with consumption substantially less than in the registration study. Treatment decisions were based on FVIII activity levels and clinical assessment. The ability to titrate rpFVIII dose using FVIII activity was considered advantageous compared with BPA therapy. Notable delays in diagnosis were observed.
authors: Bleeding & Clotting Disorders Institute, Peoria, IL, USA.
authors: M D
authors: MD
authors: Tarantino
authors: Penn Comprehensive Hemophilia and Thrombosis Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
authors: A
authors: A
authors: Cuker
authors: Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA.
authors: B
authors: B
authors: Hardesty
authors: Bleeding & Clotting Disorders Institute, Peoria, IL, USA.
authors: J C
authors: JC
authors: Roberts
authors: Department of Medicine, Division of Hematology, Department of Laboratory Medicine and Pathobiology, St Michael's Hospital, University of Toronto, Toronto, ON, Canada.
authors: M
authors: M
authors: Sholzberg
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000818:Animals; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000627:therapeutic use*; D012189:Retrospective Studies; D013552:Swine; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins
keywords: acquired haemophilia A; porcine factor VIII