OC 30.2 - Efficacy of emicizumab prophylaxis in patients with severe hemophilia A in Germany: Real-life-data documented by eDiary smart medication

Monday, July 11, 2022

11:00 AM – 11:15 AM

Location: ExCel Center, ICC Auditorium

Presentation Format: In-Person

Co-Author(s)

WM

Wolfgang Mondorf

Medical Doctor
Haemostas
Frankfurt, Hessen, Germany
HE

Hermann Eichler

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

Presenting Author(s)

Carmen Escuriola-Ettingshausen, MD

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

Co-Author(s)

RF

Ronald Fischer

Haematologist
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, Baden-Wurttemberg, Germany
HK

Holstein Katharina

physician
University Hospital Hamburg Eppendorf
Hamburg, Hamburg, Germany
Robert Klamroth, MD, PhD

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

Ulrich Sachs

Haematologist
Universitätsklinikum Gießen und Marburg
Gießen, Hessen, Germany

Background: Real-life data showing the efficacy of emicizumab treatment in patients with severe hemophilia A (PwSHA) in Germany are limited.

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

Methods: ABR, AJBR and proportion of bleed-free patients were documented using the electronic diary platform smart medication. Data of PwSHA before and after switch of treatment with FVIII concentrates to emicizumab were evaluated retrospectively. Included were patients with >24 weeks of electronic documentation after switch. Data up to and including December 2021 were analyzed.

Results: 39 PwSHA from 7 HTCs using smart medication could be included. The median age was 42 years (IQR 35.5); 81% were 18 years and older. 13 PwSHA started with electronic documentation together with the switch to emizicumab. Data from paper documentation prior to switch in these patients were not available. In 26 patients complete electronic documentation before and after switch could be evaluated. After switch to emicizumab, the mean AJBR was 0.45, the mean ABR 0.73 in all patients. In the subgroup of 26 PwSHA with a >24 weeks documentation before and after switch, the mean AJBR was 2.28 before and 0.68 after switch, the mean ABR dropped from 5.89 to 1.09 after switch. The proportion of bleeding-free patients increased from 54% before to 77% after switching to emicizumab. Despite of additional FVIII treatment in of 7 (27%) patients after the switch to emicizumab, only 2 needed additional FVIII due to joint bleeds.

Conclusion(s): Real-life data documentation by using electronic diary show a significant decrease of bleeding episodes by switching PwSHA on prophylactic treatment from FVIII concentrates to emicizumab, a more effective prophylaxis, consistent with data from clinical trials, may be discussed.