Abstract Number: PB0665
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Management of Bleeding and Trauma
Background: Since the approval of emicizumab, a bispecific, FVIII-mimetic antibody, for use in persons with congenital hemophilia A in 2018, there have been increasing case reports of off-label use of emicizumab in other bleeding disorders, including acquired hemophilia A (AHA) and von Willebrand disease (VWD).
Aims: To describe the clinical presentation and outcomes of off-label emicizumab use in AHA and VWD.
Methods: We conducted a comprehensive search in Pubmed, EMBASE, and Scopus up to January 17, 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 outcome of emicizumab use.
Results: The flowchart for the studies included in the scoping review and the primary reasons for study exclusion are described in Figure 1. Twelve studies (9 full text, 3 conference abstracts) were included in the final review, for a total of 24 patients (20 AHA, 4 VWD). Of the 20 AHA patients, 17 received emicizumab for active bleeds and 3 for surgical prophylaxis. The median duration of emicizumab use was 60 days (range: 12-180 days). There were no adverse events reported related to emicizumab use in AHA patients. All 4 VWD patients had type 3 VWD and received emicizumab for active bleeds. 3 of the 4 patients continue to be on emicizumab indefinitely, and the fourth for a total duration of 12 months. There were no adverse events reported related to emicizumab use in VWD patients. Hemostasis was achieved in all patients (AHA and VWD) in whom emicizumab was used.
Conclusions: Based on published case reports and case series, emicizumab appears to be an effective hemostatic therapy for AHA and VWD. Larger confirmatory randomized controlled trials are needed to confirm these findings.
To cite this abstract in AMA style:
Mathew Thomas V, Lim MY. Off-label Use of Emicizumab in Persons with Acquired Hemophilia A and von Willebrand Disease: A Scoping Review of the Literature [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/off-label-use-of-emicizumab-in-persons-with-acquired-hemophilia-a-and-von-willebrand-disease-a-scoping-review-of-the-literature/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/off-label-use-of-emicizumab-in-persons-with-acquired-hemophilia-a-and-von-willebrand-disease-a-scoping-review-of-the-literature/