Abstract Number: PB0466
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Basic
Background: Approximately 40-60 newborns are newly diagnosed with haemophilia A or B every year in Germany. For prevention of bleeding and sequelae including haemophilic arthropathy, the standard treatment of PUPs is prophylaxis with clotting factor concentrates, although non-factor therapies are used increasingly. The optimal time or regimen for starting prophylactic therapy to avoid the development of joint disease or neutralising antibodies to clotting factors is under debate.
Aims: In Germany, data on incidence, treatment, clinical course and outcome of newly diagnosed haemophilia patients, have not been documented yet.
Methods: The German Paediatric 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 in Germany and collects variables related to diagnosis and therapy concentrating on outcome in haemophilia, including inhibitor development, offers quality assurance and serves as a base for future studies.
Results: The database has been established on the same platform as PedNet to allow international collaborations and data analyses. Since January 1st 2017, 252 children have been enrolled from 37 participating centres. Out of these, 203 children were diagnosed with haemophilia A. The median age of diagnosis of children with severe phenotype (n=127) is 0.38 years, with moderate (n=16) 0.04 years and 1.5 years with mild phenotype (n=59). Thirty-seven children were diagnosed with haemophilia B. The median age of diagnosis of children with severe phenotype (n=25) is 0.17 years, with moderate (n=7) 2.75 years and 3.21 years with mild phenotype (n=6). By establishing new ways of data collection, such as remote documentation longitudinal documentation to collect and analyse clinical parameters can continue despite the pandemic.
Conclusions: GEPHARD has been established successfully and has adopted to the pandemic. Further results on novel treatment strategies are expected in 2021.
To cite this abstract in AMA style:
Bildingmaier C, Olivieri M, Türknetz M, Schultze-Strasser S, Escuriola Ettingshausen C, Kentouche K, Zieger B, Kurnik K, Eberl W, Königs C, GEPHARD study group . GEPHARD – The PUP Cohort of the `Standing Commission Paediatrics of the Society for Thrombosis and Haemostasis Research´ – A Progress Report [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/gephard-the-pup-cohort-of-the-standing-commission-paediatrics-of-the-society-for-thrombosis-and-haemostasis-research-a-progress-report/. Accessed September 27, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/gephard-the-pup-cohort-of-the-standing-commission-paediatrics-of-the-society-for-thrombosis-and-haemostasis-research-a-progress-report/