TY - JOUR
T1 - Safety and efficacy of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A: Results of an interventional, post-marketing study
AU - Holme, Pål André
AU - Poulsen, Lone Hvitfeldt
AU - Tueckmantel, Claudia
AU - Maas Enriquez, Monika
AU - Alvarez Román, María Teresa
AU - De Cristofaro, Raimondo
PY - 2024
Y1 - 2024
N2 - IntroductionDamoctocog alfa pegol (BAY 94-9027, Jivi (R)) is an approved extended half-life factor VIII (FVIII) for treatment of previously treated patients with haemophilia A aged >= 12 years. We report the final results of an interventional, post-marketing study of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A.MethodsIn this open-label, interventional, post-marketing, phase 4 trial (NCT04085458), previously FVIII-treated patients with severe haemophilia A aged >= 18 years received damoctocog alfa pegol for >= 100 exposure days (EDs). Patients initially received 45 IU/kg every 5 days (recommended) or 40 IU/kg twice-weekly. At Visit 3, patients' doses could be increased, or treatment frequency adapted. The primary endpoint was FVIII inhibitor development (titter >=.6 Bethesda units). Secondary endpoints included anti-polyethylene glycol (PEG) antibody development, treatment-emergent adverse events (AEs) and annualized bleeding rate (ABR).ResultsOverall, 36 patients were enrolled; 32 patients received treatment, of whom, 27 completed the study. No patients developed FVIII inhibitors; three tested transiently positive for low-titter anti-PEG antibodies without clinical relevance. Three patients reported study-drug-related AEs of mild or moderate intensity. Two patients discontinued the study due to AEs. No deaths occurred. Most patients (70%) were treated with E5D/E7D regimens. The median (Q1;Q3) total ABR (N = 30) was 3.0 (.0;9.0) pre-study and 1.8 (.7;5.9) during the study.ConclusionDamoctocog alfa pegol individualized prophylaxis regimens were well-tolerated with no immunogenicity concerns. ABRs improved following the switch from pre-study prophylaxis to damoctocog alfa pegol prophylaxis. These results support the favourable safety and efficacy profile of damoctocog alfa pegol prophylaxis.
AB - IntroductionDamoctocog alfa pegol (BAY 94-9027, Jivi (R)) is an approved extended half-life factor VIII (FVIII) for treatment of previously treated patients with haemophilia A aged >= 12 years. We report the final results of an interventional, post-marketing study of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A.MethodsIn this open-label, interventional, post-marketing, phase 4 trial (NCT04085458), previously FVIII-treated patients with severe haemophilia A aged >= 18 years received damoctocog alfa pegol for >= 100 exposure days (EDs). Patients initially received 45 IU/kg every 5 days (recommended) or 40 IU/kg twice-weekly. At Visit 3, patients' doses could be increased, or treatment frequency adapted. The primary endpoint was FVIII inhibitor development (titter >=.6 Bethesda units). Secondary endpoints included anti-polyethylene glycol (PEG) antibody development, treatment-emergent adverse events (AEs) and annualized bleeding rate (ABR).ResultsOverall, 36 patients were enrolled; 32 patients received treatment, of whom, 27 completed the study. No patients developed FVIII inhibitors; three tested transiently positive for low-titter anti-PEG antibodies without clinical relevance. Three patients reported study-drug-related AEs of mild or moderate intensity. Two patients discontinued the study due to AEs. No deaths occurred. Most patients (70%) were treated with E5D/E7D regimens. The median (Q1;Q3) total ABR (N = 30) was 3.0 (.0;9.0) pre-study and 1.8 (.7;5.9) during the study.ConclusionDamoctocog alfa pegol individualized prophylaxis regimens were well-tolerated with no immunogenicity concerns. ABRs improved following the switch from pre-study prophylaxis to damoctocog alfa pegol prophylaxis. These results support the favourable safety and efficacy profile of damoctocog alfa pegol prophylaxis.
KW - extended half-life
KW - haemophilia A
KW - prophylaxis
KW - recombinant factor FVIII
KW - extended half-life
KW - haemophilia A
KW - prophylaxis
KW - recombinant factor FVIII
UR - http://hdl.handle.net/10807/274897
U2 - 10.1111/hae.14930
DO - 10.1111/hae.14930
M3 - Article
SN - 1351-8216
VL - 30
SP - 388
EP - 394
JO - Haemophilia
JF - Haemophilia
ER -