The European Paediatric Network for Haemophilia Management ( PedNet Registry)

Recruiting

• Conditions: Factor VIII Deficiency; Factor IX Deficiency

• Registration Number: NCT02979119
• Lead Sponsor: PedNet Haemophilia Research Foundation

Brief Summary

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.

Detailed 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.

Study Timeline

• Study Start: 2014-06-01
• Study First Submitted: 2014-10-21
• Study First Submitted QC: 2016-11-28
• Study First Posted: 2016-12-01
• Status Verified: 2025-09
• Last Update Submitted: 2025-09-09
• Last Update Posted: 2025-09-16
• Primary Completion: 2039-12
• Study Completion: 2039-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 4000

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients with antibody development to exogenous clotting factors	Until patient reaches age of 18	Allo-antibodies against Factor VIII and IX; Blood test: measurement in Bethesda units (BU), positive according to local standards, for most labs \>0.5 BU

Secondary Outcome Measures

Name	Time	Method
Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.	From diagnose every 5 years until patient reaches age of 18	Effect of different prophylactic regimen on bleeding and joint damage
Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.	From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18	Effect of different ITI therapies on bleeding and joint damage. Joint damage is assessed using the HJHS and MRI.

Trial Locations

Locations (39)

Children's Health Ireland (CHI) at Crumlin; 🇮🇪 Dublin, Ireland

Universitäts-Klinik für Kinder- und Jugendheilkunde; 🇦🇹 Graz, Austria

Medical University of Vienna - Department of Paediatrics; 🇦🇹 Vienna, Austria

Service of Pediatric Haematology University Hospital Leuven; 🇧🇪 Leuven, Belgium

Division of Hematology/Oncology Hôpital St Justine; 🇨🇦 Montreal, Canada

Division of Haematology/Oncology Hospital for Sick Children; 🇨🇦 Toronto, Canada

Haemophilia Comprehensive Care Centre, Centre for Thrombosis and Haemostasis Children's University Hospital Brno; 🇨🇿 Brno, Czechia

Department of Paediatric Haematology/oncology - University Hospital Motol; 🇨🇿 Prague, Czechia

Department of Pediatrics Århus Kommunehospital Skejby Sygehus; 🇩🇰 Aarhus, Denmark

Children's Hospital Helsinki University Hospital; 🇫🇮 Helsinki, Finland

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