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Lookup NU author(s): Jan Lecouturier, Professor Neil SheerinORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0).
Background Atypical haemolytic uremic syndrome (aHUS) is a rare life-threatening disease. Lifelong treatment with intravenous eculizumab every two/three weeks was recommended but evidence is emerging that patients can stop eculizumab and restart should they relapse. However, little is known about the opinions and needs of aHUS patients on withdrawal. Objective We aimed to understand the factors that impact on 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 adults (8), and the parents of children (12), 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 regime and side effects, the time off work/school and impact 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 two 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 Understanding patient/parent experiences should guide discussions about eculizumab withdrawal. Although the availability of an alternative complement inhibitor reduces the frequency of infusions, the majority of 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. Key findings and implications Lifelong treatment can be burdensome for patients and families and have a substantial negative impact on their lives. This is particularly so when the onset of illness occurs, and treatment is commenced, in babies and young children. Long-term treatment can be disruptive to work and school, due to the fortnightly administration of eculizumab and the side effects experienced. There was a dislike for long-term medication particularly when there was uncertainty as to whether treatment was still warranted. The opportunity to consider treatment withdrawal was welcomed. Conversations between clinicians and patients/parents about withdrawal should be guided by an understanding of the experiences at onset and living on treatment, concerns and fears. Hearing parents’ and adults’ accounts of the positive impact of withdrawing from eculizumab may provide reassurance to others.
Author(s): Lecouturier J, Sheerin N
Publication type: Article
Publication status: Published
Journal: The Patient - Patient Centred Outcome Research
Year: 2025
Pages: epub ahead of print
Online publication date: 12/09/2025
Acceptance date: 21/08/2025
Date deposited: 22/09/2025
ISSN (print): 1178-1653
ISSN (electronic): 1178-1661
Publisher: Adis International Ltd.
URL: https://doi.org/10.1007/s40271-025-00771-5
DOI: 10.1007/s40271-025-00771-5
Data Access Statement: It is not possible for the data from this study to be made available.
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