Abstract Number: OC 30.2
Meeting: ISTH 2022 Congress
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.
To cite this abstract in AMA style:Mondorf W, Hermann E, Escuriola Ettingshausen C, Ronald F, Holstein K, Klamroth R, Sachs U. Efficacy of emicizumab prophylaxis in patients with severe hemophilia A in Germany: Real-life-data documented by eDiary smart medication [abstract]. https://abstracts.isth.org/abstract/efficacy-of-emicizumab-prophylaxis-in-patients-with-severe-hemophilia-a-in-germany-real-life-data-documented-by-ediary-smart-medication/. Accessed August 9, 2022.
« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-of-emicizumab-prophylaxis-in-patients-with-severe-hemophilia-a-in-germany-real-life-data-documented-by-ediary-smart-medication/