Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors' Organisation

Wall, C; Xiang, H; Palmer, B; Chowdary, P; Collins, PW; Gorman, R; Matthias, M; Percy, C; Sartain, P; Shapiro, S; Stephensen, D; Talks, K; Hay, CRM

Journal article

Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors' Organisation

Abstract:: BackgroundEmicizumab prophylaxis is restricted to severe hemophilia A in the UK. Treatment choice and safety remain a matter of debate.ObjectivesThis study was conducted to investigate factors influencing treatment choice, continued use, safety, and clinical outcomes associated with emicizumab in a national cohort of persons with severe hemophilia A without current inhibitors.MethodsA 3-year study was conducted in 618 persons with severe hemophilia A who switched to emicizumab and 413 who continued factor (F)VIII prophylaxis. Outcome measures included annualized bleed rates (ABRs), the Hemophilia Joint Health Score, and health-related quality of life.ResultsSwitchers and nonswitchers had a similar median age (26 and 28 years, respectively). Switchers had a significantly higher median (IQR) ABR than those continuing FVIII prophylaxis, but a significantly lower proportion had an inhibitor history (13.6% vs 20.5%; P = .0005). Thirty-one adverse events were reported, including 1 thrombosis (0.2%), 8/84 recurrent inhibitors (9.5%), 1 neutralizing antidrug antibody (0.2%), and 14/618 (2.3%) patients discontinued emicizumab. A higher prestudy median (IQR) ABR was observed in switchers compared with nonswitchers (2.05 [0.43, 6.06] vs 0.68 [0, 2.7]), reducing to a median (IQR) of 0 (0, 0) with emicizumab prophylaxis. The proportion with a zero-treated bleed rate increased from 35% to 71% (P = .001). An 82% reduction in bleeding into target joints was observed in favor of emicizumab. A modest (Δ= -2; P = .02) improvement in the total Hemophilia Joint Health Score was observed.ConclusionsEmicizumab selection was influenced by ABR and inhibitor history but not age. Emicizumab was generally well tolerated, with only 2.3% discontinuing the drug. A significant within-person improvement in all bleeding outcomes was observed with emicizumab.

Publication status:: Published

Peer review status:: Peer reviewed

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APA Style

Wall, C., Xiang, H., Palmer, B., Chowdary, P., Collins, P. W., Gorman, R., Matthias, M., Percy, C., Sartain, P., Shapiro, S., Stephensen, D., Talks, K., & Hay, C. R. M. (2025). Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors' Organisation. Research and Practice in Thrombosis and Haemostasis, 9(6), 103164.

MLA Style

Wall, C, et al. “Emicizumab Utilization, Safety, and Outcomes in People with Severe Hemophilia and No Inhibitors: 3-Year Follow-up. A Report from the UK Haemophilia Centre Doctors' Organisation.” Research and Practice in Thrombosis and Haemostasis, vol. 9, no. 6, 2025, p. 103164.

Chicago Style

Wall, C, H Xiang, B Palmer, et al. 2025. “Emicizumab Utilization, Safety, and Outcomes in People with Severe Hemophilia and No Inhibitors: 3-Year Follow-up. A Report from the UK Haemophilia Centre Doctors' Organisation.” Research and Practice in Thrombosis and Haemostasis 9 (6): 103164.
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