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.

Journal article

Wall, Caroline, Xiang, Hua, Palmer, Ben, Chowdary, Pratima, Collins, Peter W, Gorman, Richard, Matthias, Mary, Percy, Charles, Sartain, Paul, Shapiro, Susan, Stephensen, David, Talks, Kate and Hay, Charles 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), p. 103164. https://doi.org/10.1016/j.rpth.2025.103164
AuthorsWall, Caroline, Xiang, Hua, Palmer, Ben, Chowdary, Pratima, Collins, Peter W, Gorman, Richard, Matthias, Mary, Percy, Charles, Sartain, Paul, Shapiro, Susan, Stephensen, David, Talks, Kate and Hay, Charles R M
Abstract

Background
Emicizumab prophylaxis is restricted to severe hemophilia A in the UK. Treatment choice and safety remain a matter 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 hemophilia A without current inhibitors.

Methods
A 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.

Results
Switchers 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.

Conclusions
Emicizumab 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.

KeywordsSafety; Efficacy; Hemophilia A; Emicizumab; Non-inhibitor
Year2025
JournalResearch and Practice in Thrombosis and Haemostasis
Journal citation9 (6), p. 103164
PublisherElsevier
ISSN2475-0379
Digital Object Identifier (DOI)https://doi.org/10.1016/j.rpth.2025.103164
Official URLhttps://pmc.ncbi.nlm.nih.gov/articles/PMC12494912/pdf/main.pdf
FunderRoche Products Ltd
Publication dates
Print01 Aug 2025
Online01 Sep 2025
Publication process dates
Accepted28 Jul 2025
Deposited15 Oct 2025
Publisher's version
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Open
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