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Efficacy and Safety of BAY 94-9027 Prophylaxis for ≥5 Years: Outcomes in 36 Patients from the PROTECT VIII Extension Study

P.A. Holme1, R. Klamroth2, W. Miesbach3, M. Wang4, M.E. Mancuso5, M.T. Reding6

1Oslo University Hospital and Institute of Clinical Medicine, University of Oslo, Oslo, Norway, 2Vivantes Hospital im Friedrichshain, Berlin, Germany, 3University Hospital Frankfurt, Frankfurt, Germany, 4Bayer, Whippany, United States, 5Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy, 6University of Minnesota Medical Center, Center for Bleeding and Clotting Disorders, Minneapolis, United States

Abstract Number: PB0916

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: BAY 94-9027 is a B-domain-deleted, extended-half-life, recombinant factor VIII (FVIII) site‑specifically PEGylated with a 60 kDa polyethylene glycol. In PROTECT VIII (NCT01580293), efficacy and safety of BAY 94-9027 as treatment for previously treated severe hemophilia A patients, aged 12-65 years, were evaluated.

Aims: To examine efficacy and safety of BAY 94-9027 in patients treated with regular prophylaxis for ≥5 years from PROTECT VIII.

Methods: In PROTECT VIII, 114 patients received BAY 94-9027 prophylaxis (25 IU/kg twice weekly [2×W]) for a 10-week run-in period. Patients with ≤1 breakthrough bleed during the run-in period were randomized to receive BAY 94-9027 every 5 days (45-60 IU/kg) or every 7 days (60 IU/kg) for the 26-week study period. Patients with >1 bleed, and those eligible for randomization but enrolled after randomized arms were full, were assigned to 30-40 IU/kg 2×W. In the extension, prophylaxis patients could receive BAY 94-9027 on any PROTECT VIII regimen. Patients who switched regimen following the first seven days of the extension were assessed as a variable frequency group.

Results: At extension completion, 36 patients completed ≥5 years (median [range] 6.2 [5.0-7.0]) of BAY 94-9027 prophylaxis and a median (range) of 428.5 (293-698) exposure days. Twenty-two patients remained on the same regimen throughout the extension period. Median (Q1; Q3) annualized bleeding rate for the total prophylaxis group during extension was 1.1 (0.4; 2.1) (Figure 1). Median FVIII consumption (IU/kg/year) for prophylaxis patients was 3332 (Table 1). Five (13.9%) patients experienced non-serious treatment-emergent drug-related adverse events. No patients developed FVIII inhibitors. No deaths or thrombotic events were reported.

Conclusions: Efficacy of BAY 94-9027 was maintained over ≥5 years’ prophylaxis using infusion schedules best fitting individual patients’ needs. These data support the favorable risk-benefit profile of BAY 94-9027 and its use as a long-term treatment option for patients with hemophilia A.

  2×W (n = 4) E5D (n = 10) E7D (n = 8) VAR (n = 14) Total (N = 36)
Total dose per infusion, IU/kg, median (Q1; Q3) 35 (30; 41) 44 (43; 48) 59 (57; 62) 55 (49; 57) 51 (44; 58)
Total dose per year, IU/kg/year, median (Q1; Q3) 3725 (3011; 4262) 3179 (3122; 3695) 3083 (3015; 3286) 3826 (3547; 4269) 3332 (3144; 3991)
2×W, twice weekly; E5D, every 5 days; E7D, every 7 days; FVIII, factor VIII; VAR, variable frequency.

[Table 1: FVIII consumption in patients with ≥5 years BAY 94-9027 treatment]


[Figure 1: ABRs during the extension in patients with ≥5 years’ BAY 94-9027 treatment]

To cite this abstract in AMA style:

Holme PA, Klamroth R, Miesbach W, Wang M, Mancuso ME, Reding MT. Efficacy and Safety of BAY 94-9027 Prophylaxis for ≥5 Years: Outcomes in 36 Patients from the PROTECT VIII Extension Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/efficacy-and-safety-of-bay-94-9027-prophylaxis-for-%e2%89%a55-years-outcomes-in-36-patients-from-the-protect-viii-extension-study/. Accessed September 29, 2023.

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