Born as part of the EUHANET project, the prospective Rare Bleeding Disorders Database (PRO-RBDD) stems from the previous European Network of Rare Bleeding Disorders (EN-RBD), and collects data on patients with the most severe types of RBDs, fibrinogen and factor XIII (FXIII) deficiencies, using a prospective observational cohort design. The PRO-RBDD project aims to better identify the number of affected individuals worldwide, make use of the data collected to optimise the clinical management of patients and therapy modalities.
Thanks to the development of an international network of care providers and to the use of a web-based database, the PRO-RBDD is collecting prospective data on laboratory and clinical manifestation of disease, efficacy, and safety of treatment. Genetic characterisation of patients and external quality control assessment of laboratory assays for fibrinogen and FXIII are also carried out.
Until now 62 centres have joined PRO-RBDD (38 from the EU, 17 from Asia and Middle East, 4 from the Americas, 3 from Africa and 1 from Oceania). Of these, 19 centres are actively entering data (16 from the EU, 1 from Asia and Middle East and 2 from the Americas), and 43 centres are either pending ethics approval from their institution or having difficulties in recruiting patients.
The PRO-RBDD database currently stores data on 140 patients with fibrinogen deficiency (114 from Europe) and 64 patients with FXIII deficiency (51 from Europe).
The findings observed so far confirmed the previously reported association between fibrinogen or FXIII plasmatic levels and clinical severity of patients. Further analysis aims at establishing a cut-off level of activity capable of discriminating patients who may have spontaneous major bleedings.