Changes in Hemophilia Treatment in the Eastern Part of Germany between 2015 and 2021—Data from the Kompetenznetz Hämorrhagische Diathese Ost (KHDO)

Abstract

Introduction Treatment options for patients with hemophilia (PWH) have changed substantially in the last years. This study aimed to compare hemophilia treatment in the eastern part of Germany in 2021 with data from 2015.

Methods Substitution diaries and patient records of PWH from 2021 were collected in 13 hemophilia centers from the “Kompetenznetz Hämorrhagische Diathese Ost” (KHDO) and compared with 2015.

Results A total of 130 children and 357 adults, 411 hemophilia A (HA) and 76 hemophilia B (HB), were included in 2021, and 359 were already analyzed in 2015. In 2021, 97.8% of children and 95.7% of adults with severe hemophilia had prophylaxis compared with 98.8 and 80.2% in 2015. Plasma-derived concentrates were used in 25.6%, recombinant in 30.2%, extended half-life (EHL) factor concentrates in 24.4%, and emicizumab in 19.8% of the children with severe HA (sHA). In adults with sHA, plasma-derived, recombinant concentrates, EHL, and emicizumab were used in 21.0, 33.2, 31.2, and 14.2%, respectively. All children and 93.3% of the adults with severe HB (sHB) were on EHL. Median annual factor consumption per body weight increased in adults with sHA, remained stable in children with sHA and adults with sHB, and decreased in children with sHB between 2015 and 2021. Annualized bleeding rate (ABR) decreased in children with sHB and sHA.

Conclusion The use of EHL and emicizumab has changed hemophilia treatment. About 50% of the sHA patients switched to EHL or emicizumab and almost all sHB patients to EHL. More adults with sHA received prophylaxis and ABR decreased in children.

Zusammenfassung

Einleitung Die Behandlungsmöglichkeiten für Patienten mit Hämophilie (PMH) haben sich in den letzten Jahren erheblich verändert. Ziel dieser Studie war der Vergleich der Hämophiliebehandlung im Osten Deutschlands im Jahr 2021 mit dem Jahr 2015.

Methoden Substitutionstagebücher und Patientenakten von PMH aus dem Jahr 2021 wurden in 13 Hämophiliezentren des Kompetenznetzes Hämorrhagische Diathese Ost (KHDO) erhoben und mit Daten von 2015 verglichen.

Ergebnisse 130 Kinder und 357 Erwachsene, 411 mit Hämophilie A (HA), 76 mit Hämophilie B (HB), wurden eingeschlossen, von denen 359 bereits 2015 analysiert wurden. Im Jahr 2021 erhielten 97,8% der Kinder und 95,7% der Erwachsenen mit schwerer Hämophilie eine Prophylaxe, verglichen mit 98,8% und 80,2% im Jahr 2015. Plasmatische Faktorenkonzentrate wurden bei 25,6%, rekombinante Konzentrate bei 30,2%, Faktorenkonzentrate mit verlängerter Halbwertszeit (EHL) bei 24,4% und Emicizumab bei 19,8% der Kinder mit schwerer HA (sHA) eingesetzt. Bei Erwachsenen mit sHA wurden plasmatische Konzentrate in 21,0%, rekombinante Konzentrate in 33,2%, EHL in 31,2% und Emicizumab in 14,2% verwendet. Alle Kinder und 93,3% der Erwachsenen mit schwerer HB (sHB) erhielten EHL. Der jährliche Faktorverbrauch pro Körpergewicht stieg bei Erwachsenen mit sHA, blieb bei Kindern mit sHA und Erwachsenen mit sHB stabil und nahm bei Kindern mit sHB zwischen 2015 und 2021 ab. Die jährliche Blutungsrate (ABR) nahm bei Kindern mit sHA und sHB ab.

Schlussfolgerung Der Einsatz von EHL und Emicizumab hat die Hämophiliebehandlung verändert. Etwa 50% der sHA-Patienten wechselten zu EHL oder Emicizumab und fast alle sHB-Patienten zu EHL. Mehr Erwachsene mit sHA erhielten eine Prophylaxe und die ABR bei Kindern ist gesunken.

Keywords

hemophilia - prophylaxis - bleeding - epidemiology - factor consumption

Schlüsselwörter

Hämophilie - Prophylaxe - Blutungen - Epidemiologie - Faktorkonsum

Ethical Considerations

The study was approved by the Ethics Committee of the University of Leipzig (reference 393/22-ek) and conducted according to the Declaration of Helsinki.

^* Christian Pfrepper and Annika Siecke have contributed equally.

Publication History

Received: 20 August 2024

Accepted: 09 December 2024

Article published online:
18 February 2025

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