TY - JOUR
T1 - PROTECT VIII kids extension study
T2 - Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A
AU - Mancuso, Maria Elisa
AU - Biss, Tina
AU - Fischer, Kathelijn
AU - Maas Enriquez, Monika
AU - Steele, MacGregor
AU - Wang, Maria
AU - Tseneklidou-Stoeter, Despina
AU - Ahuja, Sanjay
AU - Kenet, Gili
N1 - Publisher Copyright:
© 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.
PY - 2021/5
Y1 - 2021/5
N2 - Introduction: BAY 94-9027 (damoctocog alfa pegol; an extended half-life PEGylated recombinant factor VIII [FVIII]) demonstrated efficacy and safety in previously treated paediatric patients (PTPs) aged <12 years with severe haemophilia A in the PROTECT VIII Kids study (NCT01775618). Aim: To evaluate the long-term safety of BAY 94-9027 in PTPs aged <12 years at enrolment. Methods: In the PROTECT VIII Kids study, boys <12 years with severe haemophilia A were enrolled in two age cohorts (6–<12 years and <6 years) and treated prophylactically twice weekly, every 5 days or every 7 days, with BAY 94-9027 for ≥50 exposure days (EDs). Patients who had completed ≥50 EDs and ≥6 months in the main study or 12-week safety expansion study were eligible to participate in the extension. Primary safety variable was frequency of inhibitor development; main efficacy variable was annualised bleeding rate (ABR). Results: Of 73 PTPs from the main/expansion studies, 59 (81%) entered the extension phase for a median (range) duration of 5.0 (0.4–5.9) years. Overall, 39 patients completed ≥5 years of treatment. No patients developed FVIII inhibitors/anti-PEG antibodies, and two patients aged <6 years discontinued. Median ABR for total bleeds was 1.5 (<6 years) and 1.9 (6–<12 years). Total ABR improved in the extension vs. the main study. In the last 12 months of treatment, median spontaneous ABR was 0.0 in both age groups. Conclusions: BAY 94-9027 showed long-term safety and efficacy for the prevention and treatment of bleeds in younger and older paediatric patients with severe haemophilia A.
AB - Introduction: BAY 94-9027 (damoctocog alfa pegol; an extended half-life PEGylated recombinant factor VIII [FVIII]) demonstrated efficacy and safety in previously treated paediatric patients (PTPs) aged <12 years with severe haemophilia A in the PROTECT VIII Kids study (NCT01775618). Aim: To evaluate the long-term safety of BAY 94-9027 in PTPs aged <12 years at enrolment. Methods: In the PROTECT VIII Kids study, boys <12 years with severe haemophilia A were enrolled in two age cohorts (6–<12 years and <6 years) and treated prophylactically twice weekly, every 5 days or every 7 days, with BAY 94-9027 for ≥50 exposure days (EDs). Patients who had completed ≥50 EDs and ≥6 months in the main study or 12-week safety expansion study were eligible to participate in the extension. Primary safety variable was frequency of inhibitor development; main efficacy variable was annualised bleeding rate (ABR). Results: Of 73 PTPs from the main/expansion studies, 59 (81%) entered the extension phase for a median (range) duration of 5.0 (0.4–5.9) years. Overall, 39 patients completed ≥5 years of treatment. No patients developed FVIII inhibitors/anti-PEG antibodies, and two patients aged <6 years discontinued. Median ABR for total bleeds was 1.5 (<6 years) and 1.9 (6–<12 years). Total ABR improved in the extension vs. the main study. In the last 12 months of treatment, median spontaneous ABR was 0.0 in both age groups. Conclusions: BAY 94-9027 showed long-term safety and efficacy for the prevention and treatment of bleeds in younger and older paediatric patients with severe haemophilia A.
KW - FVIII
KW - adolescents
KW - children
KW - damoctocog alfa pegol
KW - haemophilia A
KW - polyethylene glycol
KW - prophylaxis
UR - http://www.scopus.com/inward/record.url?scp=85102510123&partnerID=8YFLogxK
U2 - 10.1111/hae.14294
DO - 10.1111/hae.14294
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C2 - 33724632
AN - SCOPUS:85102510123
SN - 1351-8216
VL - 27
SP - 434
EP - 444
JO - Haemophilia
JF - Haemophilia
IS - 3
ER -