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Confirmed long‐term safety and efficacy of prophylactic treatment with BAY 94–9027 in severe haemophilia A: final results of the PROTECT VIII extension study
Author(s) -
Reding Mark T.,
Pabinger Ingrid,
Holme Pal Andre,
Poulsen Lone,
Negrier Claude,
Chalasani Pavani,
Maas Enriquez Monika,
Wang Maria,
Meijer Karina,
Mancuso Maria Elisa,
Lalezari Shadan
Publication year - 2021
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.14297
Subject(s) - medicine , haemophilia , prophylactic treatment , haemophilia a , term (time) , extension (predicate logic) , intensive care medicine , surgery , physics , quantum mechanics , computer science , programming language
The phase 2/3 PROTECT VIII main study demonstrated efficacy and safety of BAY 94–9027 (damoctocog alfa pegol; Jivi ® ), a B‐domain‐deleted recombinant factor VIII (FVIII), site‐specifically PEGylated to extend its half‐life. Aim To report the final efficacy and safety data for BAY 94–9027 from the PROTECT VIII extension. Methods Previously treated males aged 12–65 years with severe haemophilia A (FVIII <1%) who completed the multicentre, open‐label PROTECT VIII main study were eligible for the extension. Patients received either on demand or prophylaxis treatments (30‒40 IU/kg twice weekly [2 × W], 45‒60 IU/kg every 5 days [E5D], or 60 IU/kg every 7 days [E7D]) and could switch to any prophylaxis regimen (variable frequency) as needed. Annualised bleeding rates (ABR), zero bleeds and safety outcomes were included in this final analysis. Results At extension completion, patients (n = 121) received BAY 94–9027 for a median (range) total time of 3.9 (0.8–7.0) years. Median (Q1; Q3) total ABR was 1.49 (0.36; 4.80) for prophylaxis patients (n = 107), compared with 34.09 (20.3; 36.6) for on‐demand patients (n = 14). Median total ABRs for 2 × W (n = 23), E5D (n = 33), E7D (n = 23) and variable frequency (n = 28) groups were 1.57, 1.17, 0.65 and 3.10, respectively. Of prophylaxis patients, 20.6% were bleed‐free during the entire extension (median time, 3.2 years) and 50.0% were bleed‐free during the last 6 months. No patient developed FVIII inhibitors. No deaths or thrombotic events were reported. Conclusions Efficacy and safety of BAY 94–9027 was confirmed, with extension data supporting its use as a long‐term treatment option for patients with haemophilia A.