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.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - 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/