The PedNet Haemophilia Registry is a database owned and administered by the PedNet Foundation containing [anonymous] data of children with haemophilia. The PedNet Haemophilia Registry is physically placed in Baarn, in The Netherlands.
As of December 1st, 2016 the PedNet Registry is registered on http://ClinicalTrials.gov . ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world. The PedNet Registry is registered under the number NCT02979119
The aim of the PedNet registry is to include complete cohorts of all newly diagnosed patients born from 01-01-2000 with congenital haemophilia A and B factor VIII/ IX ≤ 0.25 IU/ dl and treated in one of the 33 participating centres. Well-defined clinical determinants of diagnosis and treatment characteristics are collected. Parents of the patients receive written and verbal information about the registry and sign informed consent before participation.
The strength of the PedNet registry is that detailed information on diagnosis, clinical signs and treatment (dose, product, bleeding, surgery) are collected. For the first 50 exposure days every reason for treatment is documented, including details on treatment. Inhibitor development is centrally defined according to pre-defined criteria. The collected data capture many clinical and laboratory and genetic parameters, which makes them suitable for a large variety of research questions. Data entry occurs throughout the year by participating centres using a web-based Case Report Form (CRF).
Currently 1972 children are included in the Registry, whereof 1241 with severe Haemophilia A and B.
Monitoring of centres is performed by external monitors. For all patients 100% of baseline data are monitored and at least 10% of follow up forms. Selection bias is prevented by inclusion of all eligible patients diagnosed in the center and implementation of strict in- and exclusion criteria.
Cohort l (2000-2009)
The first cohort of patients was born between 2000 and 2010 and was collected by 30 centres out of 16 countries participated. Detailed data of every reason for treatment, product names, etc. until 75th exposure day were collected. Gene defects are available for 95% of the patients with severe haemophilia. In order to study cohorts with sufficient follow up, the first analysis were performed on the data export of May 2011. From than onwards annual exports of the database were used for studies and publications.
The last data export of January 2018 contained for this cohort a total of n= 1208 patients, whereof n=740 were diagnosed with severe (Factor VIII/ IX activity < 0.01 IU/ml), 173 with moderate (Factor VIII/ IX activity between > 0.0 1-<0.05 IU/ml), and 295 with mild (Factor VIII/ IX activity from > 0.05 – 0.25 IU/ml) haemophilia A and B.
Cohort ll (2010-2019)
Inclusion of children born from 2010 onwards is ongoing. Currently 33 centres are participating in the Registry.
The last download of January 2018 (provided by 30 centres) contained for this cohort a total of n= 761 patients, whereof n=501 were diagnosed with severe (Factor VIII/ IX activity < 0.01 IU/ml) haemophilia A and B.
For more information on the design of the PedNet Registry click here