The European Paediatric Network for Haemophilia Management ( PedNet Registry)
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Rationale:

 Haemophilia is a rare disease; to improve knowledge international collaboration is needed.
 Well-defined clinical data will be collected from complete cohorts in order to prevent
 selection bias.

 Objective:

 To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous
 (treatment-related) determinants of inhibitor development and long term outcome.
Design: Multicenter Prospective Observational Birth Cohort Study

 Population:

 Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and
 1-1-2030.

 Intervention:

 No intervention; only documentation of patient characteristics and parameters of routine
 patient care and outcome

 Main outcome parameters:

 Outcome: clinically relevant inhibitor development, bleeding pattern and joint status on
 physical examination and imaging.

 Determinants: baseline FVIII/IX levels, measurement of inhibitory antibodies, family history,
 FVIII/IX gene mutation, details on replacement therapy (according to each infusion for the
 first 50 treatment days, and annually thereafter) and surgeries.

 Nature and extent of the burden and risks associated with participation, benefit and group
 relatedness:

 -  No burden for the patients. Well-defined clinical data will be collected from the
 medical files. Participating in this registry will not change the number of visits to
 the clinic. All outcome parameters that are collected (including laboratory results) are
 part of routine clinical care.

 -  Direct benefit is not to be expected. However, the direct interaction between centres
 that treat patients with rare diseases improves both clinical care and will result in
 better guidelines and as such may provide indirect benefit.

 -  Multicentre participation: haemophilia is a very rare condition. Therefore, collecting
 data on a multi-centre observational cohort is the only way to study this specific
 population.

 -  The registry concerns young boys with haemophilia and cannot be performed in older
 patients, as >90% of inhibitors occur develop during the first 50 exposure days, and the
 results of prophylactic replacement therapy are highly dependent on the initiation of
 this treatment.
Inclusion Criteria:

 -  Diagnosed with Haemophilia A or B

 -  Factor VIII/ IX activity of <1 to 25%

 -  Complete records of Factor treatment and bleeds

 -  Treated in one of the participating centres

 Exclusion Criteria:

 -  Patients referred because of an inhibitor*

 -  Informed consent not obtained