Which Factors Influence Decisions to Withdraw from Eculizumab: A Qualitative Study of Patients Diagnosed with aHUS.

IF 3.1 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Patient-Patient Centered Outcomes Research Pub Date : 2025-09-12 DOI:10.1007/s40271-025-00771-5

Jan Lecouturier, Neil Sheerin

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Abstract

Background: Atypical haemolytic uremic syndrome (aHUS) is a rare life-threatening disease. Lifelong treatment with intravenous eculizumab every 2/3 weeks was recommended, but evidence is emerging that many patients can stop complement inhibition and restart should they relapse. However, little is known about the opinions and needs of patients with aHUS on withdrawal.

Objective: We aimed to understand the factors that affect decisions to withdraw from treatment.

Methods: This was a qualitative study embedded in an eculizumab withdrawal trial. Data were collected through in-depth telephone interviews from January 2019 to June 2022. We conducted a thematic analysis using a constant comparative method. Interviewees included eight adults and the parents of 12 children with aHUS approached to participate in the trial. Follow-up interviews were conducted between January 2021 and June 2024 with those who withdrew from eculizumab.

Results: The onset of aHUS had been traumatic for most. Regarding eculizumab, withdrawal group participants talked of the disruptive treatment regimen and side effects, the time off work/school, and impacts on taking holidays. Decisions to withdraw from eculizumab were driven by the wish to lead more normal lives and concerns about long-term treatment. Drivers for declining withdrawal focused on relapse and its perceived impact. After 2 years, the withdrawal group had regained a semblance of normality, though fears about relapse remained, and they were aware of the need for long-term follow-up. Participants had a greater sense of control over the necessary steps should they/their child relapse.

Conclusion: An understanding of patient/parent experiences should guide discussions about eculizumab withdrawal. Although the availability of an alternative complement inhibitor reduces the frequency of infusions, most of the factors influencing withdrawal decisions remain unchanged. Support to alleviate fears in the early stages of withdrawal would be beneficial. Evidence from the main trial on successful withdrawal, and recovery time where eculizumab was reinstated, may provide reassurance to those who are uncertain about withdrawal.

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哪些因素影响退出埃曲利单抗的决定：一项诊断为aHUS患者的定性研究。

背景：非典型溶血性尿毒症综合征（aHUS）是一种罕见的危及生命的疾病。建议每2/3周静脉注射eculizumab进行终身治疗，但越来越多的证据表明，许多患者可以停止补体抑制并在复发时重新开始。然而，对aHUS患者对停药的意见和需求了解甚少。目的：我们旨在了解影响退出治疗决定的因素。方法：这是一项定性研究，嵌入在eculizumab停药试验中。从2019年1月至2022年6月，通过深度电话访谈收集数据。我们使用恒定比较法进行了主题分析。受访者包括8名成年人和12名aHUS患儿的父母，他们被邀请参加试验。在2021年1月至2024年6月期间，对退出eculizumab的患者进行了随访访谈。结果：aHUS发病多为外伤性。关于eculizumab，戒断组的参与者谈到了破坏性的治疗方案和副作用，停工/上学的时间，以及对休假的影响。决定退出eculizumab的原因是希望过上更正常的生活，以及对长期治疗的担忧。减少戒断的驱动因素主要集中在复发及其感知影响上。两年后，戒断组恢复了表面上的正常，尽管对复发的恐惧仍然存在，他们意识到需要长期随访。如果他们/他们的孩子复发，参与者对必要的步骤有更大的控制感。结论：对患者/家长经历的理解应该指导关于eculizumab停药的讨论。虽然替代补体抑制剂的可用性减少了输注的频率，但影响停药决定的大多数因素保持不变。在撤军的早期阶段支持减轻恐惧将是有益的。来自主要试验成功停药的证据，以及恢复eculizumab的恢复时间，可能为那些不确定停药的人提供安慰。

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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来源期刊

Patient-Patient Centered Outcomes Research HEALTH CARE SCIENCES & SERVICES-

CiteScore

6.60

自引率

8.30%

发文量

审稿时长

>12 weeks

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