A Real-World Evidence Analysis of the Impact of Switching from Factor VIII to Emicizumab Prophylaxis in Patients with Hemophilia A Without Inhibitors

Authors: Escobar, M; Bullano, M; Mokdad, AG; Caicedo, J; Schultz, BG; Fan, Q; Verma, S

Affiliations: Univ Texas Hlth Sci Ctr, Houston, TX 77030 USA. Takeda Pharmaceut USA, Lexington, MA USA. STATinMED, Ann Arbor, MI USA

Publication: Expert Review of Hematology; 2023

Abstract: BACKGROUND This study retrospectively compared annualized billed bleed rates (ABR(b)) in people with hemophilia A (PwHA) without inhibitors who switched from factor VIII (FVIII) prophylaxis to emicizumab. RESEARCH DESIGN AND METHODS A real-world comparison study was performed on the effect of switching from FVIII to emicizumab prophylaxis in male, non-inhibitor patients on ABR(b) using an all-payer claims database (APCD) dataset from 1 January 2014 to 31 March 2021. The identification period was from 1 November 2017 to 30 September 2020. RESULT sOne hundred and thirty-one patients were included with a total of 82 and 45 bleeds in the pre- and post-switch periods, respectively. The average follow-up period pre-switch was 978.37 days (SD 555.03), whereas the average follow-up period post-switch was 522.26 days (SD 191.36). No significant differences in mean ABR(b) were observed pre-/post-switch (0.25 and 0.20, respectively; P = 0.4456). CONCLUSIONS The results of this study demonstrate no significant reduction in ABR(b), suggesting that switching from FVIII to emicizumab may not deliver incremental benefits to PwHA receiving prophylactic care.