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 – diseases such as haemophilia A (factor VIII deficiency) and haemophilia B (factor IX deficiency), von Willebrand disease, and other rare factor deficiency diseases such as factor XI deficiency. The registry's biggest benefit is that it is a neutral point for the collection of data on the treatment of blood clotting disorders. This data can be used for research purposes. The better that patients and doctors document their treatment and concomitant circumstances, the more valuable the data is for research.
Patients' data is pseudonymised in the register and can thus be assigned to the same person year after year. Only with this approach is it possible to document long-term observations and evaluate the effects of the various therapies over a period that goes far beyond the duration of clinical studies. One example of the information that can be gained comes from data on which preparations are used and to what extent. In the future, this information can show whether certain groups of patients benefit more from certain therapies than others.
The DHR gained a legal basis in 2019 with the Law for Increased Safety in the Supply of Pharmaceuticals (Gesetz für mehr Sicherheit in der Arzneimittelversorgung). Physicians are required to report the diagnosis and treatment of patients with blood clotting disorders. Patients are obliged to document their self-treatment at home accordingly.
The Paul-Ehrlich-Institut manages the DHR in cooperation with the two major patient associations – the Haemophiliacs Interest Group (Interessengemeinschaft Hämophiler e.V., IGH) and the German Haemophilia Society (Deutsche Hämophiliegesellschaft, DHG) – and with the Society for Thrombosis and Haemostasis Research (Gesellschaft für Thrombose- und Hämostaseforschung e.V., GTH).
The DHR's data can be used for research purposes. An application for the processing of research data must be submitted to the DHR office. Our website provides transparent information on the application conditions along with the DHR's annual reports, which contain information on areas such as patient numbers and the consumption of the various medicinal products.
Physicians are required to inform their patients about the DHR and the report.
If the patient agrees to submit a report to the DHR after discussing it with their doctor, a pseudonymised data package is logged annually in the DHR. The data does not contain information on the patient's name or place of residence. The extensive data can also be used to carry out research.
If a patient does not agree to their data being collected, only a small number of datapoints are summarised in a report. This data serves to monitor consumption, i.e. the amount of medicinal product required to treat patients, in order to enable/guarantee long-term patient care. Data from more than 17,000 patients is currently available, 3,000 of which submitted pseudonymised, detailed reports.
There are a variety of therapy options for the treatment of haemophilia. The list starts with 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 recombinant manufactured 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 humanised, monoclonal antibody for the treatment of haemophilia A was authorised in an accelerated procedure by the EU Commission in 2018. This antibody is produced recombinantly and used for long-term prophylaxis. The product is approved for all age groups, however, no data is available for children under one year of age and adults over 77 years of age.
On 24 August, 2022, the EU Commission issued a conditional marketing authorisation for a gene therapy medicinal product used for the treatment of severe haemophilia A in adults with no history of factor VIII inhibitors and without detectable AAV5 antibodies. AAV5 stands for adeno-associated virus type 5. Patients form antibodies against this vector during treatment. The treatment can therefore only be carried out once and the absence of antibodies against AAV5 must be reliably demonstrated before the start of treatment.
On 20 February, 2023, the conditional marketing authorisation for the first gene therapy for the treatment of haemophilia B in adults with no history of factor IX inhibitors followed. This gene therapy product can also only be administered once.
Updated: 17.04.2024
