The European Paediatric Network for Haemophilia Management ( PedNet Registry)
- Conditions
- Factor VIII DeficiencyFactor 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 1-1-2030.
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 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 4000
- 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
- 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)
Medical University of Vienna - Department of Paediatrics
🇦🇹Vienna, Austria
Universitäts-Klinik für Kinder- und Jugendheilkunde
🇦🇹Graz, Austria
Service of Pediatric Haematology University Hospital Leuven
🇧🇪Leuven, Belgium
Division of Hematology/Oncology Hôpital St Justine
🇨🇦Montréal, 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
🇨🇿Praha, Czechia
Service Hématologique Centre Regional Traitement d'Hemophilie Bicetre
🇫🇷Le Kremlin Bicêtre, France
Service d'Hématologie Pédiatrique Hôpital Universitaire La Timone
🇫🇷Marseille Cedex-05, France
Charité Campus Virchow Klinikum, Klinik für Pädiatrie m.S. Onkologie und Hämatologie
🇩🇪Berlin, Germany
Centre de traitement des hémophiles Hôpital Universitaire Purpan
🇫🇷Toulouse, France
Department of Internal Medicine, Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain
🇩🇪Berlin, Germany
Klinik Bremen-Mitte Prof.-Hess-Kinderklinik
🇩🇪Bremen, Germany
Institut für Experimentelle Hämatologie und Transfusionsmedizin Universitätsklinikum Bonn
🇩🇪Bonn, Germany
University Hospital Frankfurt & Goethe University - Clinical and Molecular Hemostasis, Department of Pediatrics
🇩🇪Frankfurt am Main, Germany
Hämophilie Zentrum Rhein Main
🇩🇪Mörfelden-Walldorf, Germany
Dr. v. Haunersches Kinderspital University of Munich
🇩🇪Munich, Germany
The National Hemophilia Center Sheba Medical Center, Tel Hashomer
🇮🇱Ramat Gan, Israel
Haemophilia-Haemostasis Unit St. Sophia Children's Hospital
🇬🇷Athens, Greece
Azienda Ospedaliero Universitaria Careggi
🇮🇹Florence, Italy
Gaslini Hospital
🇮🇹Genova, Italy
Centro Hospitalar São João, S. Imuno-hemoterapia
🇵🇹Porto, Portugal
Van Creveld Kliniek University Medical Center Utrecht
🇳🇱Utrecht, Netherlands
A. Bianchi Bonomi Hemophilia and Thrombosis Centre IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Unitat Hemofilia Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital General Unidad de Hemofilia 1 Sur Hospitales Universitarios Virgen del Rocio
🇪🇸Seville, Spain
Unidad de Coagulopatías Hospital Universitario La Paz
🇪🇸Madrid, Spain
Department of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital
🇸🇪Stockholm, Sweden
Unidad de Coagulopatias Congenitas Hospital Universitario la Fe
🇪🇸Valencia, Spain
Lund University Hospital
🇸🇪Malmo, Sweden
Birmingham Children's Hospital NHS Trust - Department of Haematology
🇬🇧Birmingham, United Kingdom
Royal Hospital for Sick Children
🇬🇧Edinburgh, United Kingdom
Department of Haematology Royal Hospital for Sick Children
🇬🇧Glasgow, United Kingdom
Haemophila Center Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Dept of Paediatric Haematology Our Lady's Children's Hospital for Sick Children Crumlin
🇮🇪Dublin, Ireland
Inselspital Bern, University Children's Hospital
🇨🇭Bern, Switzerland
Children's Hospital Helsinki University Hospital
🇫🇮Helsinki, Finland
Oslo University Hospital
🇳🇴Oslo, Norway
Department of Pediatrics Århus Kommunehospital Skejby Sygehus
🇩🇰Aarhus, Denmark