ASH 2020: Two Real-World Perspectives on Experiences with Emicizumab Prophylaxis
Data presented at the ASH 2020 congress bring interesting insights regarding the administration of emicizumab in real clinical practice. It provides comprehensive information on the relatively high number of individuals treated with emicizumab in relation to persistence on treatment and adherence to it. Data from the EUHASS registry confirms the good safety profile of this treatment even in regular clinical practice across Europe.
Introduction
Emicizumab is a subcutaneously administered bispecific monoclonal antibody indicated for the prophylaxis of bleeding in patients with hemophilia A with or without detected inhibitors. Since it has been used in clinical practice for a relatively short time, it is interesting to observe actual adherence to this treatment as well as its safety. The results of two studies with fresh data on this issue were presented at the 62nd virtual meeting of the American Society of Hematology (ASH).
Evaluation of Adherence
A retrospective study by Californian authors evaluated data based on medication payment requests from two databases from November 2017 to December 2019.
A total of 328 patients met the required criteria. Their average age was 23 years (standard deviation ±16; range 1–64), and the average follow-up period was 8 months (245 ± 147 days). Most of the monitored patients (92%) were persistent with emicizumab prophylaxis. Among those who discontinued treatment (n = 25), the average time to discontinuation was 3 months (84 ± 124 days). However, 40% of them resumed emicizumab prophylaxis, on average within 4 months (126 ± 56 days). Adherence to emicizumab administration was high (81%), and overall 70% of patients met ≥ 80% prescribed treatment coverage.
This study on the early introduction of emicizumab into real clinical practice confirms that hemophilia patients are very adherent to this treatment and most continue it long-term. Treatment disruptions occur rather minimally, and in many cases, are only temporary. Further research should relate persistence and adherence to clinical outcomes.
Evaluation of Safety
Data presented by the European Haemophilia Safety Surveillance (EUHASS) group focused on potential new safety signals observed in real clinical practice with emicizumab treatment. The EUHASS database is managed by a large European consortium focused on pharmacovigilance.
In 2018, 148 patients with hemophilia A receiving prophylactic emicizumab were reported. Concurrent treatments included rFVIIa in 23 individuals, aPCC in 1 individual, and FVIII in 9 individuals. Only 2 potential adverse events possibly related to emicizumab administration were described. One was an acute rash reported within 48 hours of the last dose in a patient treated solely with emicizumab. The second event was a myocardial infarction described 10 hours after administering emicizumab in a patient over 65 years old who also received aPCC. No thrombotic microangiopathy or anaphylaxis was observed.
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Sources:
1. Mahajerin A., Khairnar R., Meyer C. S. et al. Real-world persistence with and adherence to emicizumab prophylaxis in persons with hemophilia A: a secondary claims database analysis. Abstract 866. 62nd ASH Annual Meeting and Exposition, 2020 Dec 5. Blood 2020; 136 (Suppl. 1): 13, doi: 10.1182/blood-2020-137632.
2. Shang A., Bienz N. S., Gadiraju R. et al. Real world safety of emcizumab: the first interim analysis in the European Haemophilia Safety Surveillance (EUHASS) database. Abstract 2685. 62nd ASH Annual Meeting and Exposition, 2020 Dec 7. Blood 2020; 136 (Suppl. 1): 29–30, doi: 10.1182/blood-2020-134905.
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