Real-world safety and effectiveness of recombinant porcine sequence factor VIII in acquired haemophilia A: A non-interventional,post-authorization safety study |
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| Authors: | Michael D. Tarantino Brandon Hardesty Ara Metjian Thomas L. Ortel Jie Chen Kayode Badejo Alice Ma Adam Cuker Anita Rajasekhar Kenneth D. Friedman Maissaa Janbain |
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| Affiliation: | 1. Bleeding and Clotting Disorders Institute, Peoria, Illinois, USA;2. Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA;3. University of Colorado, Anschutz Medical Campus, Aurora, Colorado, USA;4. Duke University, Durham, North Carolina, USA;5. Takeda Development Center Americas, Inc., Cambridge, Massachusetts, USA;6. University of North Carolina, Chapel Hill, North Carolina, USA;7. Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA;8. Department of Hematology/Oncology, University of Florida Health, Gainesville, Florida, USA;9. Medical College of Wisconsin and Versiti/Blood Center of Wisconsin, Milwaukee, Wisconsin, USA;10. Tulane School of Medicine, New Orleans, Louisiana, USA |
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| Abstract: | Introduction Recombinant porcine factor VIII (rpFVIII, susoctocog alfa) is indicated for the treatment of bleeding episodes in adults with acquired haemophilia A (AHA). Aim To provide long-term real-world safety and effectiveness data for rpFVIII in the management of AHA bleeding episodes. Methods US PASS (NCT02610127) was a multicentre, uncontrolled, open-label, post-marketing safety surveillance study conducted in adults with AHA. Data were collected retrospectively or prospectively for 180 days after rpFVIII treatment. The primary outcome was the incidence of treatment-related serious adverse events (SAEs). Secondary outcomes included haemostatic effectiveness of rpFVIII and rpFVIII utilization. Results Fifty-three patients were enrolled from December 2015 to June 2019 (prospective, n = 30; retrospective, n = 23). Six patients experienced seven treatment-related SAEs (incidence 12.0%). The most common treatment-related SAE was FVIII inhibition (inhibiting antibodies to rpFVIII; incidence 8.0%, 95% CI: 2.2–19.2). Five patients reported seven thromboembolic events; one was an SAE and possibly related to rpFVIII. Of bleeding events treated with rpFVIII, 80.3% (57/71) of bleeds resolved with rpFVIII. The median (range) dose of rpFVIII per infusion was 50 (10–300) units/kg, with a median (range) of 6.0 (1–140) infusions and a median (range) time from bleed onset to bleed resolution of 14.0 (2.0–132.7) days. Conclusion In this real-world study of rpFVIII for AHA, no new safety signals were identified compared with previous clinical trial findings. Eighty percent of bleeds resolved with rpFVIII treatment. |
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| Keywords: | acquired haemophilia A effectiveness long-term data real-world study recombinant porcine FVIII safety |
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