Objectives: In Germany ~40–60 newborns are diagnosed with haemophilia A or B per year. Haemophilia leads to recurrent bleeds resulting in increased morbidity and mortality. Prophylaxis with clotting factor concentrates is the standard of care to prevent bleeds and sequela including haemophilic arthopathy. The optimal timing or regimen to start prophylaxis to avoid the development of joint disease or neutralizing antibodies to clotting factors are still being discussed. Except for the German haemophilia database documenting a limited set of data, no data are available on incidence, treatment, clinical course and outcome of newly diagnosed haemophilia patients in Germany.
Methods: The German Pediatric Haemophilia Research Database (GEPHARD) includes all children and adolescents (< 18 years) that have been diagnosed with haemophilia A or B (FVIII or FIX levels < 25%) after January 1st, 2017. This prospective registry is open to all centres and documents variables related to diagnosis and therapy. The registry concentrates on outcome in haemophilia, including inhibitor development, offers quality assurance and serves as a base for future studies.
Results: The database has been established and will be used on the same platform as PedNet to allow international collaborations and data analyses. Funding has been secured for 5 years. In the first 21st months, 102 children and adolescents were enrolled from 24 participating centres. 91 children were diagnosed with haemophilia A including 47, 11 and 33 with a severe, moderate or mild phenotype, respectively. 11 children were diagnosed with haemophilia B including 7, 2 and 2 with a severe, moderate or mild phenotype, respectively. Longitudinal documentation is being initiated to document and analyze clinical parameters and outcome.
Conclusions: GEPHARD has been established and is well accepted in the community with more than 100 children already included. After having resolved administrative and contracting challenges, longitudinal documentation is starting in a large number of newly diagnosed, previously untreated children in Germany.
