Koenigs, Christoph and Bidlingmaier, Christoph and Aumann, Volker and Behnisch, Wolfgang and Buehrlen, Martina and Chada, Martin and Halimeh, Susan and Heine, Sabine and Heller, Christine and Holzhauer, Susanne and Holzschuh, Linus and Horneff, Silvia and Huetker, Sebastian and Kentouche, Karim and Kim, Sorah and Klamoth, Robert and Knoefler, Ralf and Kramer, Sonja and Kurnik, Karin and Oldenburg, Johannes and Olivieri, Martin and Schultze-Strasser, Stephan and Tuerknetz, Mira and Vaillant, Vera and Wiegering, Verena and Wieland, Ivonne and Zierk, Jakob and Eberl, Wolfgang and Laws, Hans-Juergen and Sigl-Kraetzig, Michael and Wermes, Hannover Cornelia and Zieger, Barbara and Demir, Eren and Hassenpflug, Wolf and Schober, Sarah and Escuriola-Ettingshausen, Carmen and Sparber-Sauer, Monika (2022) A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany - First Data from the GEPHARD Study Group. HAMOSTASEOLOGIE, 42. S24-S31. ISSN 0720-9355, 2567-5761
Full text not available from this repository. (Request a copy)Abstract
Objectives Initial treatment in patients with haemophilia remains challenging. The choice of therapy, timing, dose and frequency have been and are still under intense debate. New treatment options like novel factor concentrates and non-factor therapies broaden the discussion. Design The German Paediatric Haemophilia Research Database (GEPHARD) is a multicentre prospective observational study including children and adolescents with haemophilia A or B (FVIII or FIX levels < 25 IU/dL) in a German treatment centre after January 1st, 2017. A cross-sectional analysis was performed in June 2021. Results 249 children and adolescents from 22 participating centres in Germany were analysed in this cross-sectional analysis. 203 patients suffered from haemophilia A (PwHA) and 46 from haemophilia B (PwHB). The median age at diagnosis for Pw severe HA or HB was 6 or 2 months, the median age at analysis was 33 or 35 months for Pw severe HA or B, respectively. 117 Pw severe HA received treatment, including plasma derived concentrates (n = 43), standard recombinant concentrates (n = 23), extended half live concentrates (n = 33) and non-replacement therapies (n = 18). For Pw severe HB, plasma derived concentrates (n = 3), standard recombinant concentrates (n = 8) and extended half live concentrates (n = 14) were used. Current inhibitors were reported in 16 PwHA and 1 PwHB. Conclusions GEPHARD was successfully established as a national cohort for newly diagnosed PwH in Germany. Epidemiological and treatment data were presented. Longitudinal analyses of this growing cohort will allow to value treatment strategies and their outcome in the evolving treatment landscape.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | IMMUNOLOGICAL DANGER SIGNALS; FACTOR-VIII PRODUCTS; INHIBITOR DEVELOPMENT; HEMOPHILIA; REGIMEN; haemophilia; previously untreated patients; PUPs; Inhibitors; Cohort |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 01 Feb 2024 06:47 |
| Last Modified: | 01 Feb 2024 06:47 |
| URI: | https://pred.uni-regensburg.de/id/eprint/58609 |
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