Efficacy of emicizumab in patients with severe haemophilia A without factor VIII inhibitors in Germany: evaluation of real-life data documented by the smart medication eDiary

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Carmen Escuriola Ettingshausen - , Hämophilie-Zentrum Rhein Main (HZRM ) GmbH (Autor:in)
  • Wolfgang Eberl - , Städtisches Klinikum Braunschweig gGmbH (Autor:in)
  • Hermann Eichler - , Universitätsklinikum des Saarlandes (Autor:in)
  • Ronald Fischer - , SRH Kurpfalzkrankenhaus Heidelberg. (Autor:in)
  • Christina Hart - , Universitätsklinikum Regensburg (Autor:in)
  • Katharina Holstein - , Universitätsklinikum Hamburg-Eppendorf (UKE) (Autor:in)
  • Ralf Knöfler - , Klinik und Poliklinik für Kinder- und Jugendmedizin (Autor:in)
  • Jürgen Kreutz - , Chugai Pharma Germany GmbH (Autor:in)
  • Caspar David Kühnöl - , Universitätsklinikum Halle (Autor:in)
  • Wolfgang A Miesbach - , Universitätsklinikum Frankfurt (Autor:in)
  • Christian Pfrepper - , Universitätsklinikum Leipzig (Autor:in)
  • Andreas Rösch - , Smart Medication eHealth Solutions GmbH (Autor:in)
  • Ulrich J Sachs - , Universitätsklinikum Gießen und Marburg GmbH (Autor:in)
  • Karolin Trautmann-Grill - , Medizinische Klinik und Poliklinik I (Autor:in)
  • Wolfgang Mondorf - , HAEMOSTAS | Labor für Hämostaseologie Frankfurt (Autor:in)

Abstract

BACKGROUND: Systematically documented data on real-world use of emicizumab, a bispecific antibody factor (F)VIII mimetic, are still lacking in people with severe haemophilia A (PwSHA). Smart medication, a real-time, online platform, monitors treatment administration and outcomes for people with haemophilia A in Germany.

OBJECTIVE: To evaluate annualised bleeding rates (ABRs) and annualised joint bleeding rates (AJBRs), using data documented in the smart medication eDiary, for PwSHA receiving emicizumab.

DESIGN: Data for 97 PwSHA without FVIII inhibitors who started emicizumab treatment between 1 January 2018 and 31 March 2023, with >24 weeks of documentation after switching from FVIII replacement, were collected in the smart medication eDiary. Those with ⩾24 weeks of pre-emicizumab data were included for analysis 24 weeks before and after switching.

METHODS: The primary objective was to evaluate ABR and AJBR for treated bleeds. The proportion of bleed-free participants was calculated and administration frequency for FVIII and emicizumab were collected. The mean dosing frequencies for FVIII replacement and emicizumab were also evaluated.

RESULTS: The mean calculated ABR and AJBR were 0.64 and 0.39, respectively, after initiating emicizumab. For those with documentation before starting emicizumab (n = 58), ABR decreased by 79.6% and AJBR decreased by 90.8%. The proportion of bleed-free participants increased by 21.3%, and joint bleed-free participants increased by 18.2%. The median FVIII dosing frequency was every 3.5 days (n = 54; range: 1.0-20.8); median emicizumab dosing frequency was every 11.2 days (N = 97; range: 6.6-29.4).

CONCLUSION: Real-world data collected using the smart medication eDiary provide insights into efficacy outcomes after switching from FVIII replacement to emicizumab prophylaxis. Bleeds, including joint bleeds, decreased after switching.

Details

OriginalspracheEnglisch
Seitenumfang17
FachzeitschriftTherapeutic Advances in Hematology
Jahrgang15
Frühes Online-Datum29 Nov. 2024
PublikationsstatusVeröffentlicht - Dez. 2024
Peer-Review-StatusJa

Externe IDs

PubMedCentral PMC11607772
Scopus 85211573202