Withdrawing from Eculizumab: interview study of patients’ views
Research type
Research Study
Full title
Eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome: embedded qualitative study exploring patient perspectives
IRAS ID
237008
Contact name
Neil Sheerin
Contact email
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
This study is part of a larger trial to explore whether patients with kidney disease, called atypical haemolytic uraemic syndrome, can safely withdraw from the drug Eculizumab. The study employs qualitative methods and the aim is to explore the views of patients/parents on withdrawing, on the drug itself and the monitoring plan that is part of the trial. We wish to interview those who agree (up to 25) and decline (up to 20) to withdraw from Eculizumab.
Patients/parents will be asked about the qualitative study by recruitment staff at sites when they are approached to take part in the withdrawal trial. They will be given an information sheet about the qualitative study and reply sheet to record their name and contact number if they wish to be approached to discuss taking part in an interview. This can be handed back to the recruitment staff or alternatively they can take the information home and return the slip in a pre-paid envelope.
Once the patient/parent has agreed to be contacted the researcher will telephone to discuss the qualitative study and answer any questions they have. If they wish to participate, a date and time will be arranged to conduct the telephone interview. Consent will be taken just prior to the interview. A topic guide will be developed with the input of the PPI group supporting this study and wider team. A sub-set (up to 20) of patients/parents in the withdrawal group will be asked if they agree to be approached about a follow up interview at end of withdrawal period or if they relapse.
Participants will be asked if they would like to receive a short report of the findings of the interview study when it is complete. For advice on wider dissemination the team will consult the PPI group.
Lay summary of study results: Two key factors impacted on decisions to withdraw from eculizumab. The first relates to their initial illness and diagnosis (or suspected diagnosis) of aHUS, the changed existence from rarely being ill and no experience of hospital to living with the ‘ticking time bomb’ threat of a potential relapse, constantly watching for anything they feel may act as a trigger and monitoring any changes in their regular tests. The second was adjusting to the disruption of frequent long-term treatment, the sideeffects, and the limitations some experienced physically and socially. Although only a small number of participants declined withdrawal, their decision was influenced by the perceived disruption of a potential relapse and the view that treatment was not burdensome. For withdrawal participants their key motivations were to try to attain some normalcy in their lives and concerns with being on long-term medication – medication that may not be warranted. Monitoring and being able to quickly reinstate eculizumab – and knowing it is effective – should they or their child relapse provided reassurance.
Similar findings on reasons for withdrawal/non-withdrawal of medication have been reported in studies conducted with patients/caregivers with juvenile idiopathic arthritis rheumatoid arthritis.
Discussions about withdrawal from eculizumab should be based upon, and guided by, an understanding of patients’ experiences of the onset of the illness. Evidence from the main study on the optimum monitoring, successful withdrawal, and recovery time where eculizumab was reinstated may provide reassurance to those who are uncertain about withdrawal.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0113
Date of REC Opinion
2 May 2018
REC opinion
Favourable Opinion