SETS aHUS: Stopping Eculizumab Treatment Safely in aHUS
Research type
Research Study
Full title
Multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome
IRAS ID
235552
Contact name
Neil Sheerin
Contact email
Sponsor organisation
THE NEWCASTLE UPON TYNE HOSPITALS NHS FOUNDATION TRUST
Eudract number
2017-003916-37
Duration of Study in the UK
3 years, 2 months, 30 days
Research summary
Lifelong treatment with Eculizumab is currently recommended for atypical Haemolytic Uraemic Syndrome (aHUS) due to risk of relapse. Many physicians are now questioning the recommendation for indefinite treatment and feel alternative treatment strategies should be considered based upon the following:
Lack of evidence
Data prior to the availability of Eculizumab suggests that if patients survive the initial episode of aHUS with intact renal function, less than 50% of patients will subsequently relapse. Therefore, over half of patients receiving maintenance Eculizumab treatment could be doing so unnecessarily.
Burden of Treatment
Withdrawal of drug would lead to a reduction in the burden of treatment (intravenous Eculizumab injections every 2 weeks and use of prophylactic antibiotics), replacing it with reduced monitoring after a period of intense monitoring. Patients will also avoid potentially fatal complications associated with Eculizumab use (6 of approximately 100 patients treated have suffered severe complications – death, meningococcal or other unexpected, severe infection). There would be cost savings for the NHS of over £10.6 million if 50% of study participants withdraw for the 2 years of the study.
Risk of unsupervised withdrawal
Of 10 UK patients identified who have withdrawn from Eculizumab because of patient/physician choice, only 1 patient has required re-initiation of treatment. Relapse may occur after withdrawal of treatment but early re-initiation of treatment should avoid harm to patients.
As the number of patients on Eculizumab increases, so does potentially unnecessary treatment, cost to the NHS and burden of treatment to patients and their families. Therefore, a tested method of safe withdrawal and monitoring will be vital for efficient delivery of this high cost treatment.The aim of the current trial is to establish an evidence based treatment strategy for patients with aHUS that includes safe withdrawal of treatment and the reintroduction of Eculizumab in those patients who relapse.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0078
Date of REC Opinion
13 Apr 2018
REC opinion
Further Information Favourable Opinion