Journal article
Recombinant B-domain-deleted porcine sequence factor VIII (r-pFVIII) for the treatment of bleeding in patients with congenital haemophilia A and inhibitors
Haemophilia : the official journal of the World Federation of Hemophilia, Vol.23(1), pp.33-41
01/01/2017
DOI: 10.1111/hae.13108
PMID: 27761964
Abstract
Introduction: Development of inhibitors to human FVIII (hFVIII) significantly complicates the control of bleeding events in patients with haemophilia A. Aim: This prospective, multicentre, open-label, non-comparative, Phase II study evaluated the haemostatic activity of a recombinant B-domain-deleted porcine FVIII (r-pFVIII), in the treatment of non-life/ non-limb-threatening bleeding in individuals with haemophilia A and FVIII inhibitors. Methods: Acute bleeding episodes in patients with pFVIII inhibitor titres < 0.8 BU mL(-1) were treated with 50 U kg(-1) body weight r-pFVIII. Those with pFVIII inhibitor titres of > 0.8 BU mL(-1) received an initial calculated r-pFVIII loading dose followed by 50 U kg(-1) treatment dose. Treatment continued at 6-hourly intervals until bleeding was determined, controlled or till a maximum of eight doses was reached. Results: All 25 bleeding episodes in nine patients (mean age: 23.7 years; range: 14-34 years) were controlled successfully with eight or fewer injections of r-pFVIII. The median time from bleeding onset to the administration of r-pFVIII was 5.7 h (range: 1.5-20.0 h). Twenty of the bleeding episodes (80%) were controlled with one treatment dose of r-pFVIII (with or without a loading dose, median dose: 200.8 U kg(-1); range: 50-576 U kg(-1)) regardless of pFVIII level. r-pFVIII was well tolerated and no treatment-emergent serious adverse events were considered by the investigator to be related to r-pFVIII administration. Conclusion: The results suggest that FVIII replacement therapy with r-pFVIII could be a viable alternative to bypassing agents for the treatment of bleeding episodes in individuals with haemophilia A and FVIII inhibitors.
Details
- Title: Subtitle
- Recombinant B-domain-deleted porcine sequence factor VIII (r-pFVIII) for the treatment of bleeding in patients with congenital haemophilia A and inhibitors
- Creators
- J. N. Mahlangu - University of the WitwatersrandT. A. Andreeva - City Haemophilia Center, Saint Petersburg, Russia.D. E. Macfarlane - University of IowaC. Walsh - Icahn School of Medicine at Mount SinaiN. S. Key - University of North Carolina at Chapel Hill
- Resource Type
- Journal article
- Publication Details
- Haemophilia : the official journal of the World Federation of Hemophilia, Vol.23(1), pp.33-41
- Publisher
- Wiley
- DOI
- 10.1111/hae.13108
- PMID
- 27761964
- ISSN
- 1351-8216
- eISSN
- 1365-2516
- Number of pages
- 9
- Grant note
- Inspiration Biopharmaceuticals Inc., Laguna Niguel, CA, USA
- Language
- English
- Date published
- 01/01/2017
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984359831502771
Metrics
10 Record Views