The European Paediatric Network for Haemophilia Management ( PedNet Registry)

PedNet Haemophilia Research Foundation

Status: Enrolling

Conditions: Factor VIII Deficiency; Factor IX Deficiency

Study type: Observational

Funder types: Other

Identifiers

NCT02979119

Version 6.4 November 2022

Details and patient eligibility

About

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.

Full description

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 1-1-2040.

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 and girls 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.

Enrollment: 4,000 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

4,000 participants in 4 patient groups

Cohort I

Description:

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX \<1%) haemophilia A or B, born from January 1st 2000 until December 31st 2009 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort II

Description:

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX \<1%) haemophilia A or B, born from January 1st 2010 until December 31st 2019 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort III

Description:

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX \<1%) haemophilia A or B, born from January 1st 2020 until December 31st 2029 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort IV

Description:

Cohort IV Group/Cohort Description: Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX \<1%) haemophilia A or B, born from January 1st 2030 until December 31st 2039 who have been or are to be treated with coagulation proteins in one of the participating centres