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 We analysed in-depth telephone interviews thematically using a constant comparative method. Interviewees included adults (8), and the parents of children (12), with aHUS approached to participate in an eculizumab withdrawal trial.

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. 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.

The authors have declared no competing interest.

This study was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme.

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A favourable ethical opinion was obtained from North East Tyne & Wear South Research Ethics (18/NE/0113)

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