Eculizumab withdrawal and monitoring in atypical haemolytic uraemic syndrome (SETS aHUS): a multicentre, open label, prospective, single arm trial.

ABSTRACT:

Background

Atypical Haemolytic Uraemic Syndrome is a rare disease, associated with high morbidity and mortality. Eculizumab, a monoclonal complement inhibitor, is an effective treatment but the optimal way to use this high-cost medication has not been determined. The SETS aHUS trial aimed to establish the safety of eculizumab withdrawal and the effectiveness of a monitoring protocol to detect disease relapse and reintroduction of treatment if relapse occurs.

Methods

The SETS aHUS multicentre, open label, prospective, single-arm trial enrolled participants from 15 UK hospitals. Patients over two years of age with aHUS who were receiving eculizumab therapy for at least six months were eligible to withdraw from treatment, replacing it with monitoring to assess disease activity

SUBMITTER: Bryant A 

PROVIDER: S-EPMC12811780 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature