PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A

Maria Elisa Mancuso, Tina Biss, Kathelijn Fischer, Monika Maas Enriquez, MacGregor Steele, Maria Wang, Despina Tseneklidou-Stoeter, Sanjay Ahuja, Gili Kenet

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)434-444
JournalHaemophilia
Volume27
Issue number3
DOIs
Publication statusPublished - 2021

Keywords

  • adolescents
  • children
  • damoctocog alfa pegol
  • FVIII
  • haemophilia A
  • polyethylene glycol
  • prophylaxis

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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