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- Title
Off‐label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: A scoping review of the literature.
- Authors
Thomas, Vinay Mathew; Abou‐Ismail, Mouhamed Yazan; Lim, Ming Y.
- Abstract
Introduction: Since the approval of emicizumab, a bispecific, factor VIII‐mimetic antibody, for use in persons with congenital haemophilia A in 2018, there have been increasing case reports and case series of off‐label use of emicizumab in other bleeding disorders, including acquired haemophilia A (AHA) and von Willebrand disease (VWD). Aim: We conducted a scoping review on the use of emicizumab in AHA and VWD, focusing on the clinical presentation and outcomes. Methods: We conducted a comprehensive search in PubMed, EMBASE and Scopus up to July 15, 2021. The following criteria were applied to the studies identified in the initial search: patients had a diagnosis of AHA or VWD; and the study reported on the clinical outcome of emicizumab use. Results: Seventeen studies were included in the final review for a total of 41 patients (33 AHA, eight type 3 VWD). The majority of AHA patients and all type 3 VWD patients were started on emicizumab for active/recurrent bleeds. The dosing regimen of emicizumab used varied significantly in AHA patients. All patients had a clinical response to emicizumab use. One AHA patient developed a stroke on emicizumab use in association with concomitant recombinant FVIIa use for surgery. Data on adverse events from emicizumab use were not specifically reported in 24.4% of patients (four AHA, six type 3 VWD). Conclusion: Based on published case reports and case series, emicizumab appears to be an effective haemostatic therapy for AHA and VWD. Larger confirmatory clinical trials are needed to confirm these findings.
- Subjects
VON Willebrand disease; EMICIZUMAB; HEMOPHILIACS; OFF-label use (Drugs); LITERATURE reviews
- Publication
Haemophilia, 2022, Vol 28, Issue 1, p4
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.14450