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

Lookup NU author(s): Dr Tina Biss

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This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0).

Abstract

© 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd. 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.

Publication metadata

Author(s): Santagostino E, Kenet G, Fischer K, Biss T, Ahuja S, Steele M

Publication type: Article

Publication status: Published

Journal: Haemophilia

Year: 2020

Volume: 26

Issue: 3

Pages: e55-e65

Print publication date: 01/05/2020

Online publication date: 25/03/2020

Acceptance date: 26/02/2020

Date deposited: 06/04/2020

ISSN (print): 1351-8216

ISSN (electronic): 1365-2516

Publisher: Wiley-Blackwell

URL: https://doi.org/10.1111/hae.13963

DOI: 10.1111/hae.13963

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