The „German Pediatric Hemophilia Research Database“ (GEPHARD) – a project of the `Standing Committee Pediatrics of the Society for Thrombosis and Hemostasis Research´ to improve the quality of hemophilia care for children
C. Bidlingmaier1, C. Escuriola Ettingshausen2, K. Kentouche3, M. Olivieri1, W. Eberl4, B. Zieger5, K. Kurnik1, C. Königs6, for the GEPHARD Study Group (1Munich, Germany, 2Mörfelden, Germany, 3Jena, Germany, 4Braunschweig, Germany, 5Freiburg, Germany, 6Frankfurt Main, Germany)
Pediatric and neonatal thrombosis and hemostasis
Time: 17:15 - 18:15
Objective: Hemophilia is a rare disease, diagnosed in 40-60 children in Germany per year. As a chronic hemorrhagic disease it leads to recurrent bleeds resulting in severe morbidity. Prophylaxis with clotting factor concentrates is the standard of care for bleeding prevention, but when and how to start is unclear. Strategies to avoid the development of neutralizing antibodies are strongly discussed. Exact documentation of any kind of treatment related factors in a large cohort is necessary to collect outcome data and to improve hemophilia care. Currently two registries are open for German patients: The German Hemophilia Registry (DHR) collects data from all centers focusing on patient numbers and clotting factor use. Naturally, being a registry for all hemophilia patients, the DHR is not specific on pediatric issues. In contrast, the European PedNet registry focuses on children. However, only 5 German hemophilia treatment centers (HTC) are part of the PedNet group and therefore, PedNet data do not reflect the situation in Germany.
Methods: To investigate German treatment modalities and outcome data in newly diagnosed hemophilia patients the Standing Committee Pediatrics of the Society for Thrombosis and Hemostasis Research (GTH) is establishing a multicenter, prospective, observational registry. It will capture all replacement therapy related variables in children with factor VIII or IX levels of < 1 to 25%. Additionally, the registry will serve as a base for future investigational activities e.g. on immunology, outcome and imaging. Participating centers can get support in case of medical questions and documentation, not only for the GEPHARD registry but also the DHR.
Results: GEPHARD is patronized by the GTH. A steering committee and a commission consisting of representatives from 18 HTC have been established. The protocol is currently undergoing institutional review for ethical and data security issues. GEPHARD is registered at clinicaltrails.gov (NCT02912143) and the Registry of Patient Registries (RoPR ID: 11758) and will start enrollment in 01/2017.
Conclusion: The new German Pediatric Hemophilia Research Database (GEPHARD), which is open to all German HTCs, will offer a base to investigate any kind of treatment related research questions in children with hemophilia all over Germany. Cooperation with existing registries is intended.