Premium
PROTECT VIII Kids: BAY 94‐9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A
Author(s) -
Santagostino Elena,
Kenet Gili,
Fischer Kathelijn,
Biss Tina,
Ahuja Sanjay,
Steele MacGregor
Publication year - 2020
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.13963
Subject(s) - medicine , haemophilia a , haemophilia , bleed , clinical endpoint , pediatrics , clinical trial , surgery
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.