Emicizumab utilisation, safety and outcomes in people with severe haemophilia and no inhibitors: Three years follow-up. A report from the UK Haemophilia Centre Doctors’ Organisation

Wall, Caroline, Xiang, Hua, Palmer, Ben, Chowdary, Pratima, Collins, Peter W. ORCID: https://orcid.org/0000-0002-6410-1324, Gorman, Richard, Matthias, Mary, Percy, Charles, Sartain, Paul, Shapiro, Susan, Stephensen, David, Talks, Kate, Hay, Charles R.M., Bagot, Catherine, Chowdary, Pratima, Clark, Amanda, Creagh, Desmond, Dolan, Gerry, Farrelly, Cathy, Gooding, Richard, Grainger, John, Grimley, Charlotte, Hanley, John, Kazmi, Rashid, Maclean, Rhona, Mangles, Sarah, Motwani, Jayashree, Page, Andrew, Payne, Jeanette, Rayment, Rachel, Symington, Emily, Talksm, Kate and Thomas, Will 2025. Emicizumab utilisation, safety and outcomes in people with severe haemophilia and no inhibitors: Three years follow-up. A report from the UK Haemophilia Centre Doctors’ Organisation. Research and Practice in Thrombosis and Haemostasis , 103164. 10.1016/j.rpth.2025.103164

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Abstract

Background Emicizumab prophylaxis is restricted to severe haemophilia A in the UK. Treatment choice, and safety remain matters of debate. Objectives This 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 haemophilia A (PwSHA) without current inhibitors. Methods A three-year study was conducted in 618 PwSHA who switched to emicizumab and 413 who continued FVIII prophylaxis. Outcome measures included annual bleed-rates (ABR), Haemophilia Joint Health Score (HJHS) and health-related quality of life (HRQoL). Results Switchers and non-switchers had a similar median age (26 and 28 years). Switchers had a significantly higher median (IQR) ABR than those continuing FVIII prophylaxis but a significantly lower proportion with an inhibitor history (13.6 vs 20.5%, p=0.0005)). Thirty-one adverse events were reported, including one thrombosis (0.2%), 8/84 recurrent inhibitors (9.5%) and one neutralising anti-drug antibody (0.2%) and 14/618 (2.3%) patients discontinued emicizumab. A higher pre-study median (IQR) ABR was observed in switchers compared to non-switchers (2.05 (0.43,6.06) versus 0.68 (0,2.7), reducing to a median (IQR) of 0 (0,0) with emicizumab prophylaxis. The proportion with zero-treated bleed-rate increased from 35% to 71% (P=0.001). An 82% reduction in bleeding into target joints was observed in favour of emicizumab. A modest (Δ=-2, P=0.02) improvement in total HJHS was observed. Conclusions Emicizumab selection was influenced by ABR and inhibitor history but not age. Emicizumab was generally well tolerated, only 2.3% discontinuing the drug. A significant within-person improvement in all bleeding outcomes was observed with emicizumab.

Item Type:	Article
Date Type:	Published Online
Status:	In Press
Schools:	Schools > Medicine
Publisher:	Elsevier
ISSN:	2475-0379
Date of Acceptance:	28 July 2025
Last Modified:	15 Sep 2025 14:00
URI:	https://orca.cardiff.ac.uk/id/eprint/181095

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