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

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2025-08-01 DOI:10.1016/j.rpth.2025.103164

Caroline Wall , Hua Xiang , Ben Palmer , Pratima Chowdary , Peter W. Collins , Richard Gorman , Mary Matthias , Charles Percy , Paul Sartain , Susan Shapiro , David Stephensen , Kate Talks , Charles R.M. Hay , UK Haemophilia Centre Doctors’ Organisation (UKHCDO)

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引用次数: 0

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.

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Emicizumab在无抑制剂的严重血友病患者中的使用、安全性和结局：3年随访一份来自英国血友病中心医生组织的报告

背景：在英国，demicizumab预防仅限于严重血友病A。治疗选择和安全性仍然是一个有争议的问题。目的：本研究旨在调查在全国无当前抑制剂的严重a型血友病患者中影响emicizumab治疗选择、持续使用、安全性和临床结果的相关因素。方法一项为期3年的研究对618名改用emicizumab的严重血友病A患者和413名继续使用因子(F)VIII预防的患者进行了研究。结果测量包括年出血率（abr）、血友病关节健康评分和与健康相关的生活质量。结果转换者和非转换者的中位年龄相似（分别为26岁和28岁）。切换者的中位（IQR） ABR明显高于继续进行FVIII预防的患者，但具有抑制剂史的比例明显较低（13.6% vs 20.5%; P = 0.0005）。报告了31例不良事件，包括1例血栓形成（0.2%），8/84例复发性抑制剂（9.5%），1例中和性抗药物抗体（0.2%），14/618例（2.3%）患者停用emicizumab。与非切换组相比，切换组的研究前中位（IQR） ABR更高（2.05 [0.43,6.06]vs 0.68[0,2.7]），使用emicizumab预防后中位（IQR）降至0（0,0）。出血率为零的比例从35%增加到71% （P = 0.001）。emicizumab可使靶关节出血减少82%。观察到血友病关节健康总分有适度改善（Δ= - 2; P = .02）。结论单抗的选择受ABR和抑制剂史的影响，而不受年龄的影响。Emicizumab总体耐受性良好，只有2.3%的患者停药。使用emicizumab观察到所有出血结果的显著改善。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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