aHUS: Atypical Haemolytic Uraemic Syndrome

Publication of the oral iptacopan therapy in patients with C3 glomerulopathy: a randomised, double-blind, parallel group, multicentre, placebo-controlled, phase 3 study
The oral iptacopan APPEAR-C3G trial, led by Professor David Kavanagh, has been published here in The Lancet.
This trial looked at the safety and efficacy of iptacopan vs. placebo in patients with C3 glomerulopathy.
The results from this clinical trial are promising, showing it was an effective treatment demonstrating a favourable safety profile.
Until now there has been no effective treatment for this condition and most patients progressed to end-stage kidney failure. This treatment has been shown to stop protein loss from the kidneys and stabilise kidney function.
Publication of the Stopping Eculizumab Treatment Safely in aHUS (SETS aHUS) Trial
The SETS aHUS trial, led by Professor Neil Sheerin, has been published here in The Lancet Regional Health Europe.
This trial looked at whether Eculizumab could be stopped in some aHUS patients without causing any harm to them.
The results from this clinical trial are very encouraging, demonstrating that it was as safe to stop treatment as continuing with Eculizumab, provided patients were adequately monitored and knew what to do in the case of suspected aHUS relapse.
We are currently in discussion with NHS England about what the trial findings mean for aHUS patients, and we will be in touch with all of our patients on treatment over the next few months to discuss the trial, and what the results mean for them.
