Prospective, Observational Study of the Clinical Outcomes of FVIII Treatment in Adults and Adolescents with Severe Haemophilia A.

ABSTRACT:

Objective

To assess real-world treatment patterns and outcomes in previously treated patients ≥12 years old with severe haemophilia A treated with marketed factor VIII (FVIII) replacement products.

Methods

Data were collected prospectively between 25 January 2019 and 30 November 2020 across 45 sites in 17 countries. Primary endpoint was annualized bleed rate (ABR). Secondary endpoints included factor consumption, bleed treatment, joint health, and safety. Exploratory endpoints included pain and quality of life outcomes.

Results

A total of 157 patients received ≥1 FVIII injection (prophylaxis n  = 139, on-demand n  = 19). Mean (standard deviation; SD) observation period was 43.1 (13.3) weeks. Median (quarter [Q]1, Q3) ABR was 2.0 (0.0, 5.7) for those on p

SUBMITTER: Chowdary P 

PROVIDER: S-EPMC12223957 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature