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DOI: 10.1055/s-0042-1757552
A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany – First Data from the GEPHARD Study Group
Funding This work was supported by unrestricted grants from Bayer, Biotest, CSL Behring, Grifols, Intersero, Novo Nordisk, Pfizer, Sobi, Takeda
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.
Zusammenfassung
Ziele Der Therapiebeginn bei neu diagnostizierten Patienten mit Hämophilie stellt eine Herausforderung dar. Der beste Zeitpunkt, das optimale Therapieregime und die verwendeten Medikamente waren und sind Gegenstand intensiver Diskussionen. Die Einführung neuer Therapieformen hat die Diskussion weiter verstärkt.
Design Die German Paediatric Haemophilia Research Database (GEPHARD) rekrutiert Kinder und Jugendliche bis 18 Jahre mit einer ab dem 01. Januar 2017 neu diagnostizierten Hämophilie A oder B und einer Restaktivität unter 25 IU/dL. Es werden Daten zu Diagnose, Therapie und Outcome erfasst. Im Juni 2021 wurde eine Querschnittsanalyse durchgeführt.
Ergebnisse Daten von 249 Patienten aus 22 Einrichtungen konnten in einer Querschnittstudie analysiert werden. Davon haben 203 Personen eine Hämophilie A (PwHA) und 46 eine Hämophilie B (PwHB). Der Altersmedian bei Diagnosestellung war für jeweils für die schwere Form bei PwHA 6 und bei PwHB 2 Monate, bei Auswertung jeweils 33 und 35 Monate. 117 PwHA mit schwerer Form erhielten zum Zeitpunkt der Analyse eine Therapie, mit plasmatischen (n = 43), rekombinanten (n = 23), halbwertzeitverlängerten, rekombinanten Faktorpräparaten (n = 33) und “Nicht-Faktor”-Präparaten (n = 18). Patienten mit schwerer HB nutzten plasmatische (n = 3), klassische rekombinante (n = 8) oder halbwertzeitverlängerte Präparate (n = 14). Inhibitoren wurden zum Zeitpunkt der Analyse bei 16 PwHA und 1 PwHB gemeldet.
Zusammenfassung GEPHARD konnte erfolgreich als nationale Kohortenstudie neu diagnostizierter, bislang nicht behandelter Patienten mit Hämophilie etabliert werden. In der aktuellen Querschnittsanalyse konnten Daten zu aktuellen Therapieformen ausgewertet werden. Zukünftige, longitudinale Analysen der Kohorte werden weitere Daten zu Behandlungsstrategien, insbesondere auch zum Therapierfolg unter den sich wandelnden Therapieformen liefern.
Publication History
Received: 28 November 2021
Accepted: 22 August 2022
Article published online:
26 October 2022
© 2022. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)
Georg Thieme Verlag KG
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