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

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.

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

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.

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.

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.