PB0643 - Efficacy of emicizumab prophylaxis in patients with severe hemophilia A in Germany: Follow-up evaluation of real-life-data documented by smart medication eDiary

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Co-Author(s)

WM

Wolfgang Mondorf, MD

Medical Doctor
HZRM
Moerfelden-Walldorf, Hessen, Germany

Co-Author(s)

Hermann Eichler, MD (he/him/his)

Professor of Transfusion Medicine and Haemostaseology
Institute of Clinical Haemostaseology and Transfusion Medicine, University and University Hospital of the Saarland, Homburg, Germany
Homburg, Saarland, Germany
RF

Ronald Fischer

Haematologist
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, Baden-Wurttemberg, Germany
Katharina Holstein, MD (she/her/hers)

physician
University Hospital Hamburg Eppendorf
Hamburg, Hamburg, Germany
Robert Klamroth, MD, PhD (he/him/his)

Priv. Doz. Dr. med.
Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology
Berlin, Berlin, Germany
Wolfgang Miesbach, MD

Professor and Head of Coagulation Disorders and Comprehensive Care Centre
University Hospital Frankfurt, Frankfurt, Germany
Frankfurt, Hessen, Germany
HR

Heinrich Richter

Haematologist
Hämophiliezentrum Münster
Münster, Nordrhein-Westfalen, Germany
KT

Karolin Trautmann

Haematologist
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, Sachsen, Germany
Ulrich Sachs, PhD

Physician
Institute for Clinical Immunology, Transfusion Medicine and Hemostasis, Justus-Liebig-University, Langhansstr. 7, 35392 Giessen, Germany.
Giessen, Hessen, Germany

Presenting Author(s)

Carmen Escuriola-Ettingshausen, MD

Physician
Haemophilia Centre Rhine-Main, Mörfelden-Walldorf, Germany
Mörfelden-Walldorf, Hessen, Germany

Background: Systematically documented data on real-world use of emicizumab prophylaxis in patients with severe haemophilia A (PwSHA) in Germany are still lacking. We present real-life data on efficacy of emicizumab in PwSHA across German Haemophilia Treatment Centres (HTCs) as of Dec 31, 2022.

Aims: The aim of the study was to find out whether a change in ABR or AJBR could be detected in the real-world data of smart medication eDiary after switching patients from FVIII to emicizumab.

Methods: ABR, AJBR and proportion of bleed-free patients were documented using the electronic diary platform smart medication. Data of PwSHA 24 weeks before and after switch from FVIII to emicizumab prophylaxis were evaluated.

Results: 88 pediatric and adult PwSHA from HTCs in Germany in could be included. The median age was 35 years (IQR 35); 71% were 18 years and older. 44 PwSHA started with electronic documentation when switched to emizicumab. In 44 patients with electronic documentation before and after switch could be evaluated. All PwSHA were on FVIII prophylaxis at 1 - 5 daily intervals. After switching to emicizumab prophylaxis, the mean AJBR was 0.30 and the mean ABR 0.78 in all patients. In the subgroup of 44 PwSHA with documentation before and after switch, the mean AJBR dropped significantly from 2.08 before to 0.40 after switch (p < 0.01) and the mean ABR of all bleeds from 3.19 to 1.20. The proportion of joint- patients with zero AJBR increased from 64% with FVIII prophylaxis to 91% with emicizumab prophylaxis. Despite of additional FVIII treatment in 39% of patients after the switch to emicizumab (e.g. during loading phase), only 6 (7%) needed additional FVIII due to joint bleeds.

Conclusion(s): The real life-data show a significant decrease of bleeding episodes after switching to emicizumab prophylaxis and confirm the favorable data from previous clinical trials.