World Haemophilia Day is celebrated annually on 17 April. Haemophilia is a rare hereditary disease that leads to a blood clotting disorder. There are mild, moderate and severe forms. The genetic disease mainly occurs in men and causes the blood of affected patients to clot slowly or not at all. There are various forms of haemophilia, most notably haemophilia A and B. The blood of individuals with these conditions lacks one of the proteins required for blood coagulation (coagulation factors). There are a variety of therapy options for the treatment of haemophilia. The Paul-Ehrlich-Institut, the Federal Institute for Vaccines and Biomedicines, is actively committed to ensuring the quality, efficacy and safety of the various therapy options for haemophilia.
A wide variety of therapy options are available to haemophilia patients. At the top of the list are the plasma factor products from blood and plasma donation that have been established for decades. Since the 1990s, plasma factor products have been supplemented by manufactured recombinant factor products that no longer require blood donation. In addition, recombinant factor products with an extended half-life have been on the market since 2015. A monoclonal antibody for the treatment of haemophilia A was also authorised for the first time in the EU in 2018. Gene therapeutics for the treatment of haemophilia A and B were first authorised in 2022 and 2023. Two other monoclonal antibodies have recently received their marketing authorisations. Both are authorised for the treatment of both haemophilia A and haemophilia B.
The German Haemophilia Registry (Deutsches Hämophilieregister, DHR) is an online database that collects data on the diagnosis, medical history, and treatment of patients with blood clotting disorders. The registry's biggest benefit is that the data is collected at a neutral point and it can also be used for research purposes. The Paul-Ehrlich-Institut manages the DHR in cooperation with the two large patient associations – the Haemophiliacs Interest Group (Interessengemeinschaft Hämophiler e.V., IGH) and the German Haemophilia Society (Deutsche Hämophiliegesellschaft, DHG) – as well as the Society for Thrombosis and Haemostasis Research (Gesellschaft für Thrombose und Hämostaseforschung, GTH).
Updated: 17.04.2025
