Abstract Number: PB0779
Meeting: ISTH 2020 Congress
Background: Antihemophilic factor VIII (recombinant), porcine sequence (susoctocog alfa, OBI-1, BAX 801, TAK-672; Baxalta US Inc., a Takeda company, Lexington, MA, USA; recombinant porcine FVIII [rpFVIII]) has low cross-reactivity with anti-human FVIII antibodies, and can be used to treat bleeding episodes (BEs) in adults with acquired hemophilia A (AHA).
Aims: This ongoing post-authorization study assesses the safety (primary objective), utilization, and effectiveness of rpFVIII in clinical practice.
Methods: This is an interim analysis of a multicenter, non-controlled, open-label, single-cohort, observational, non-interventional study in the EU (EUPAS16055; NCT03199794). Patients with AHA aged ≥18 years who had received or were receiving rpFVIII for BEs (initial and subsequent events) as per clinical practice were retrospectively or prospectively identified and enrolled. Patients with known anaphylactic reaction to rpFVIII, excipients, or hamster protein were excluded. Adverse events (AEs)/serious AEs were reported up to 180 days after the last dose of rpFVIII. Additional data collected included BEs and rpFVIII treatment, bleeding control, and inhibitor levels.
Results: As of June 3, 2019, 18 patients (12 males; mean±SD age, 75.2±8.8 years) were enrolled (median follow-up [n=8], 203.5 days). Serious AEs (none related to rpFVIII) were reported in 6/18 patients (cardiac failure, death, multiple organ dysfunction syndrome, sudden cardiac death, atypical pneumonia, syncope, and hemorrhage). No thromboembolic event occurred. Physician-assessed AEs of special interest (drug resistance [lack of effect], n=1; FVIII inhibition [anti-rpFVIII antibodies], n=2) were serious. Of the initial BEs (12 spontaneous, 5 traumatic, 1 surgical), 14 resolved (median [range]: 7.5 [1-49] days) and 3 remained unresolved following a mean±SD (median; range) rpFVIII loading dose of 69.8±50.9 (51.5; 32-250 U/kg).
Conclusions: In this interim analysis, these real-world data demonstrate an acceptable safety and effectiveness profile for rpFVIII in controlling bleeds in patients with AHA, and provide insights into how rpFVIII use may be optimized to personalize therapy.
To cite this abstract in AMA style:Miesbach W, Ambaglio C, Knoebl P, Lodigiani C, Santoro R, Chen J, Tripkovic N, Klamroth R. Real-World Use of Recombinant B-Domain-Deleted, Porcine-Sequence Factor VIII in Patients with Acquired Hemophilia A: An Interim Analysis of the Post-Authorization Safety Study in the European Union (EU) [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/real-world-use-of-recombinant-b-domain-deleted-porcine-sequence-factor-viii-in-patients-with-acquired-hemophilia-a-an-interim-analysis-of-the-post-authorization-safety-study-in-the-european-union-e/. Accessed November 30, 2020.
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