BAY 81-8973 prophylaxis in Patients with Hemophilia A: Interim Usage and Safety Results from the TAURUS Real-World Study

C. Santoro¹, M. Wang², B. Fuh³, P. Maes⁴, M.E. Mingot-Castellano⁵, R. Berrueco⁶, E. Detering⁷, J.F. Cabre Marquez⁸, P.Q. Lê⁹

¹Hematology, Policlinico Umberto I Hospital, Rome, Italy, ²University of Colorado, Aurora, United States, ³East Carolina University, Greenville, United States, ⁴University Hospital of Antwerp, Edegem, Belgium, ⁵Regional University Hospital Carlos Haya, Division of Hematology, Málaga, Spain, ⁶Sant Joan de Déu Children's Hospital, Barcelona, Spain, ⁷Bayer, Berlin, Germany, ⁸Bayer, Mexico City, Mexico, ⁹Queen Fabiola Children's University Hospital, Brussels, Belgium

Abstract Number: PB0870

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: BAY 81-8973 (Kovaltry^®) is an unmodified, full length, recombinant factor VIII (FVIII) indicated for prophylaxis and treatment of bleeds in patients with hemophilia A. Safety and efficacy were established in the LEOPOLD clinical trials.

Aims: To highlight interim findings on the real-world usage and safety of BAY 81-8973 from the TAURUS study.

Methods: TAURUS (NCT02830477) is an international, open-label, prospective, non-interventional study enrolling patients of all ages with moderate/severe hemophilia A (FVIII ≤5% or ≤1%) and ≥50 exposure days to any FVIII, receiving BAY 81‑8973 prophylaxis in standard clinical practice. This interim analysis reports switching behavior and safety.

Results: At data cutoff (June 2019), 264 patients had received ≥1 prophylactic dose of BAY 81-8973. The median age at baseline was 25.0 years (97.3% were previously treated with FVIII). 191 patients fulfilled inclusion criteria and had ≥1 follow-up assessment after receiving BAY 81-8973. At baseline, 60.7% received prophylaxis >2.5×/week and 36.6% received prophylaxis ≤2.5×/week (data missing for 5 patients). Three patients switched from ≤2.5×/week at baseline to >2.5×/week at cutoff; eight patients switched from >2.5×/week to ≤2.5×/week. The most frequent reason for the initial switch to BAY 81-8973 was “physician decision” (61.8%); reasons for initial dose/dosing frequency are shown in

Figure 1. The median prescribed weekly dose was 56.9 IU/kg.
The safety population included 263 patients. 22.1% experienced an adverse event (AE): all were treatment emergent and none were related to inhibitor development or positive inhibitor measurement. One AE (pruritis in one patient) was drug-related and led to discontinuation of BAY 81-8973. No other AEs were drug-related. Serious AEs were reported in 20 patients (7.6%), but none were drug-related.

[Figure 1: Reasons for initial dose/dosing frequency]

Conclusions: Data analyzed in this interim report confirm a reassuring safety profile and indicate that prophylaxis regimens before and after initiation of BAY 81-8973 were similar and remained stable during the treatment period.

To cite this abstract in AMA style:

Santoro C, Wang M, Fuh B, Maes P, Mingot-Castellano ME, Berrueco R, Detering E, Cabre Marquez JF, Lê PQ. BAY 81-8973 prophylaxis in Patients with Hemophilia A: Interim Usage and Safety Results from the TAURUS Real-World Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/bay-81-8973-prophylaxis-in-patients-with-hemophilia-a-interim-usage-and-safety-results-from-the-taurus-real-world-study/. Accessed May 18, 2021.

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