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Observational Study
. 2022 Oct;42(S 01):S24-S31.
doi: 10.1055/s-0042-1757552. Epub 2022 Oct 26.

A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany - First Data from the GEPHARD Study Group

GEPHARD Investigators; Contributors:; Contributors to the cross-sectional analysis:; Study commission
Collaborators, Affiliations
Observational Study

A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany - First Data from the GEPHARD Study Group

GEPHARD Investigators et al. Hamostaseologie. 2022 Oct.

Erratum in

Abstract
in English, German

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.

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.

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Conflict of interest statement

Christoph Königs and Christoph Bidlingmaier report All support for the present manuscript from Bayer Healthcare, Biotest, CSL Behring, Grifols, Intersero, Novo Nordisk, Pfizer, Sobi, and Takeda; Consulting fees from Bayer, Roche/Chugai, Sobi/Sanofi, Takeda, and CSL Behring; Payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from BFSH, Novo Nordisk, Sobi/Sanofi, and CSL Behring Support for attending meetings and/or travel from Biotest and Sobi; Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid from Co-chair Standing Committee Haemophilia of the GTH.

Figures

Fig. 1
Fig. 1
Age at diagnosis. The age of diagnosis in months of PUPs (n = 249) diagnosed in 2017-2021 is shown according to the severity of haemophilia A (a) or B (b).
Fig. 2
Fig. 2
Choice of concentrates in severe haemophilia A and B. The use of FVIII or FIX concentrates or non-replacement therapy (NRT) is shown in percentage for PwHA (n = 117) (a) or PwHB (n = 25) (b) receiving treatment at the time of the cross-sectional analysis.
Fig. 3
Fig. 3
Current concentrate use according to year of diagnosis. The FVIII or FIX concentrates or non-replacement therapy (NRT) used at the time of this cross-sectional analysis (June 2021) are shown in percentages according to the year of diagnosis.
See this image and copyright information in PMC

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