Hamostaseologie 2019; 39(S 01): S1-S92
DOI: 10.1055/s-0039-1680122
SY12 Paediatric Haemophilia
Georg Thieme Verlag KG Stuttgart · New York

GEPHARD - the PUP Cohort of the `Standing Commission Paediatrics of the Society for Thrombosis and Haemostasis Research´ - A Progress Report

C. Bidlingmaier
1   Dr. von Hauner Children's Hospital, LMU Munich, Pediatric Hemophilia Centre, München, Germany
,
M. Olivieri
1   Dr. von Hauner Children's Hospital, LMU Munich, Pediatric Hemophilia Centre, München, Germany
,
C. Escuriola-Ettingshausen
2   HZRM Hämophilie Zentrum Rhein Main GmbH, Mörsfelden, Germany
,
K. Kentouche
3   Department of Pediatric Hematology and Oncology, University Hospital Jena, Jena, Germany
,
W. Eberl
4   Children's Hospital of Braunschweig, Braunschweig, Germany
,
B. Zieger
5   Department of Pediatrics and Adolescent Medicine, University Medical Center Freiburg, Freiburg, Germany
,
K. Kurnik
1   Dr. von Hauner Children's Hospital, LMU Munich, Pediatric Hemophilia Centre, München, Germany
,
C. Königs
6   Department of Pediatric and Adolescent Medicine, University Hospital Frankfurt, Frankfurt, Germany
,
GEPHARD Study Group› Author Affiliations
Further Information

Publication History

Publication Date:
13 February 2019 (online)

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    Objectives: In Germany ~40–60 newborns are diagnosed with haemophilia A or B per year. Haemophilia leads to recurrent bleeds resulting in increased morbidity and mortality. Prophylaxis with clotting factor concentrates is the standard of care to prevent bleeds and sequela including haemophilic arthopathy. The optimal timing or regimen to start prophylaxis to avoid the development of joint disease or neutralizing antibodies to clotting factors are still being discussed. Except for the German haemophilia database documenting a limited set of data, no data are available on incidence, treatment, clinical course and outcome of newly diagnosed haemophilia patients in Germany.

    Methods: The German Pediatric Haemophilia Research Database (GEPHARD) includes all children and adolescents (< 18 years) that have been diagnosed with haemophilia A or B (FVIII or FIX levels < 25%) after January 1st, 2017. This prospective registry is open to all centres and documents variables related to diagnosis and therapy. The registry concentrates on outcome in haemophilia, including inhibitor development, offers quality assurance and serves as a base for future studies.

    Results: The database has been established and will be used on the same platform as PedNet to allow international collaborations and data analyses. Funding has been secured for 5 years. In the first 21st months, 102 children and adolescents were enrolled from 24 participating centres. 91 children were diagnosed with haemophilia A including 47, 11 and 33 with a severe, moderate or mild phenotype, respectively. 11 children were diagnosed with haemophilia B including 7, 2 and 2 with a severe, moderate or mild phenotype, respectively. Longitudinal documentation is being initiated to document and analyze clinical parameters and outcome.

    Conclusions: GEPHARD has been established and is well accepted in the community with more than 100 children already included. After having resolved administrative and contracting challenges, longitudinal documentation is starting in a large number of newly diagnosed, previously untreated children in Germany.


     

    No conflict of interest has been declared by the author(s).