A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany - First Data from the GEPHARD Study Group.

ABSTRACT:

Objectives

 Initial treatment in patients with haemophilia remains challenging. The choice of therapy, timing, dose and frequency have been and are still under intense debate. New treatment options like novel factor concentrates and non-factor therapies broaden the discussion.

Design

 The German Paediatric Haemophilia Research Database (GEPHARD) is a multicentre prospective observational study including children and adolescents with haemophilia A or B (FVIII or FIX levels <25 IU/dL) in a German treatment centre after January 1st, 2017. A cross-sectional analysis was performed in June 2021.

Results

 249 children and adolescents from 22 participating centres in Germany were analysed in this cross-sectional analysis. 203 patients suffered from haemophilia A (PwHA) and 46

SUBMITTER: GEPHARD Investigators 

PROVIDER: S-EPMC9794454 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature