Abstract Number: PB0917
Meeting: ISTH 2020 Congress
Background: BAY 94-9027 (damoctocog alfa pegol; Jivi®) is a B-domain-deleted recombinant factor VIII (FVIII), site-specifically PEGylated to extend its half-life. The phase 2/3 PROTECT VIII study (NCT01580293) demonstrated the efficacy and safety of BAY 94-9027 as prophylactic and on-demand therapy for adults with severe hemophilia A. Reducing the frequency of joint bleeds is expected to reduce arthropathy and improve quality of life in hemophilia A.
Aims: To investigate the long-term efficacy of BAY 94-9027 prophylaxis, particularly on joint bleeding outcomes, in patients with hemophilia A.
Methods: Prophylaxis patients who completed the PROTECT VIII study could enter the optional extension study. Prophylactic treatment regimens included twice per week, every five days (E5D) and every seven days (E7D). Patients who switched regimens during the extension study were assessed in the variable frequency group (VAR). Annualized bleeding rates (ABR) and joint ABR were assessed in this final analysis.
Results: Median (range) time in extension for patients on prophylaxis during the extension (n=107) was 3.2 (0.1-6.3) years. Median (Q1; Q3) ABR was 8.0 (2.0; 16.0) prestudy (76.6% received prophylaxis prior to enrollment) and joint ABR was 5.0 (1.0; 12.0). Median (Q1; Q3) total ABR in the extension was 1.5 (0.4; 4.8) and joint ABR was 0.9 (0.0; 3.4) (Figure 1). The overall spontaneous median ABR during the extension was 0.8 (0.0; 2.9). Throughout the extension, 20.6% of prophylaxis patients had 0 total bleeds; 29.9% had 0 joint bleeds. In the last six months of the extension (n=96), 50.0% and 58.3% of patients had 0 total and joint bleeds, respectively (Figure 2).
Conclusions: Data from this completed phase 2/3 study and its extension show that BAY 94‑9027 prophylaxis is efficacious in adults with severe hemophilia A at dose intervals of up to E7D and results in a low incidence of joint bleeds with long-term use for several years.
To cite this abstract in AMA style:Reding MT, Santagostino E, Poulsen LH, Kerlin BA, Wang M, Di Minno MND. Joint Bleeding Outcomes in Patients with Hemophilia A Receiving Long-Term Prophylaxis with BAY 94-9027 in the PROTECT VIII Extension Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/joint-bleeding-outcomes-in-patients-with-hemophilia-a-receiving-long-term-prophylaxis-with-bay-94-9027-in-the-protect-viii-extension-study/. Accessed June 22, 2021.
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