PROTECT VIII Kids: BAY 94‐9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A

INTRODUCTION: BAY 94‐9027, a site‐specifically PEGylated, B‐domain‒deleted recombinant factor VIII (FVIII) with extended half‐life, demonstrated efficacy for bleed prevention and treatment in previously treated adolescents and adults with severe haemophilia A. AIM: To assess BAY 94‐9027 in children with severe haemophilia A. METHODS: In the two‐part PROTECT VIII Kids study, boys <12 years with <1% FVIII and >50 exposure days (EDs) to FVIII were enrolled in two cohorts (<6 years; 6‐<12 years) and treated with BAY 94‐9027 prophylaxis twice‐weekly, every 5 days, or every 7 days at physician discretion for ≥50 EDs (Part 1) or twice‐weekly for 12‐weeks (Part 2). Annualized bleeding rate (ABR) was a primary efficacy endpoint; FVIII inhibitor development was the primary safety variable. RESULTS: At study completion, 25 patients had been treated twice‐weekly, 28 in the every‐5‐day group, and 8 in the every‐7‐day group. Median ABR for all bleeds was 2.9 (Part 1) and 2.4 (Part 2) and similar in younger and older patients; median ABR for joint bleeds was 0 for both cohorts. In the last 90 days’ treatment, median ABR was 0 for younger and older patients (Part 1). Of 149 reported bleeds, 93% were treated with ≤2 infusions. Twelve patients, the majority <6 years (n = 11), discontinued due to apparent loss of efficacy or hypersensitivity. No FVIII inhibitors developed. CONCLUSIONS: In PROTECT VIII Kids, which allowed tailoring of prophylaxis to individual clinical response, BAY 94‐9027 was efficacious for bleed prevention and treatment in previously treated children with severe haemophilia A.