The prospective Rare Bleeding Disorders Database (PRO-RBDD) is continuing to collect data on patients with fibrinogen and factor XIII (FXIII) deficiencies using a prospective observational cohort design.
Recently the results of the analysis of patients with FXIII deficiency were presented at the Annual meeting of International Society of Thrombosis and Haemostasis-SSC held in Montpellier in May 2016. Results on 64 patients showed that FXIII coagulant activity is correlated with clinical severity defined as age at first bleeding, age at diagnosis and severity of bleeding history. In addition, the minimum hemostatic FXIII level that should be achieved to prevent spontaneous major bleeding was also reported.
Moreover, both yearly bleeding incidence and cumulative incidence was reduced in patients on prophylaxis compared with patients on on-demand therapy observed for a median follow up period of 528 days (IQR: 790-185). This allows us to strongly recommend prophylaxis in patients with FXIII deficiency to prevent bleeding episodes.
The second part of the project is focused on Fibrinogen deficiency and results on the prospective evaluation of 96 patients has been recently submitted to the upcoming America Society of Hematology congress.
Finally two external quality control assessment exercises of laboratory assays for fibrinogen and FXIII were also carried out. Results were shown during the Annual Congress of International Society of Thrombosis and Haemostasis held in Amsterdam in June 2013 and have been recently submitted for publication.
We invite new partners to join the project in order to enlarge our study populations on both Fibrinogen and FXIII deficiencies and to fulfil all gaps in the management of these two coagulation disorders.
For more information on how to join the project, you can visit the website http://eu.rbdd.org.