Early withdrawal of a treatment for patients with a rare kidney disease is possible without relapse, safer for patients and saves the NHS millions of pounds, new research has revealed.
Atypical Haemolytic Uraemic Syndrome (aHUS) is a life-threatening condition caused by an uncommon defect in the immune system which, in the past, led to kidney failure.
Newcastle Hospitals, working with Newcastle University, supported clinical trials into pioneering drug, eculizumab, which led to the NHS approving the treatment from 2015, positively transforming the outlook for patients with the disease.
Now a new study from researchers from Newcastle Hospitals and Newcastle University, funded by the National Institute for Health and Care Research (NIHR) and published today in The Lancet Regional Health Europe, found most patients can stop eculizumab after six months and their disease will not relapse.
Careful monitoring by medical experts allows the drug to be stopped, with the potential to restart the treatment if the disease returns.
The withdrawal of eculizumab is not only of huge benefit to patients’ health and wellbeing but also represents considerable savings to the NHS of £4.2 million per patient over their lifetime.
‘Free from treatment burden’
Neil Sheerin, professor of nephrology at Newcastle University and consultant nephrologist at The Newcastle Upon Tyne Hospitals NHS Foundation Trust, led the study.
He said: “Our findings are exciting as they have the potential to significantly change the way we manage aHUS and this may be life-changing for some patients.
“Initially, when a patient started treatment for aHUS they faced a lifetime of eculizumab and the dangers associated with it.
“Now we have shown that many people can stop the treatment, freeing them from the burden of regular intravenous injections and removing risk of serious infection associated with the drug.
“In addition, significant efficiency savings for the NHS of £110.4 million over five years could be realised in this patient population through more targeted use of this high-cost treatment.”
When first approved by the National Institute for Health and Care Excellence, it was recommended that patients stay on eculizumab for the rest of their lives unless there was a medical reason to stop.
However, with the treatment, patients have a 500 to 1,000-fold increased risk of meningococcal sepsis and this is immediately eliminated with its withdrawal.
Professor Sheerin added: “What we now have is data to show the withdrawal of eculizumab is safe and effective after an initial six months – this is a huge step forwards in our understanding of the treatment.”
Findings of study
The study involved 28 participants, aged 2 to 59 from England and Scotland, who had been on eculizumab for at least six months. It was led by the renal research team at the Freeman Hospital.
All patients had their treatment withdrawn. Only four participants’ kidney disease returned and the remaining 24 have remained aHus free to date.
Professor Anthony Gordon, Programme Director for the NIHR Health Technology Assessment (HTA) Programme, which funded the trial, said: “Two key priorities for the NIHR are finding the most safe and effective treatments to help people live better, healthier lives for longer, and identify how care can be delivered in the most cost-effective way.
“The results from this trial show why embedding clinical research within the NHS is so important. Not only do they highlight that eculizumab can be safely withdrawn over time for the majority of aHUS patients, reducing the risk of adverse effects, they also show that potential savings can be made, making the NHS more efficient.”
Experts at the National Renal Complement Therapeutics Centre, a collaboration between Newcastle University and Newcastle Hospitals, will continue to monitor patients closely and adapt treatment pathways according to the evidence they generate.
Professor Sheerin said: “This will allow us to answer important questions, such as can people withdraw again from eculizumab once they’ve relapsed, and can we predict more accurately which people will have a relapse following treatment withdrawal?”
Louise’s story
Louise Percival knows first-hand the benefits of being taken off eculizumab.
The 35-year-old was diagnosed with aHus in 2017 and was on the drug until she had it withdrawn as part of the study.
Louise suffered a number of negative side effects from eculizumab, such as regular migraines, hair loss and breathlessness that impacted her daily life.
The pension claims handler, from York, said: “When I was diagnosed with aHUS I felt overwhelmed as it was a huge burden to live with – it was life-changing as it’s something that will never go away.
“I strongly disliked the eculizumab infusions as they had to be given religiously every two weeks, which impacted my holidays and social events.
“I’m a very active person, enjoying hiking and the gym, so the side effects and time needed for the treatment were detrimental to my quality of life.
“Not to mention I have a fear of needles, so having a cannula put in my arm every fortnight was very distressing.”
Louise has successfully stopped eculizumab with no relapses, significantly improving her quality of life.
She said: “All of the side effects disappeared and I feel like myself again.
“Initially, I was told eculizumab would be a lifelong commitment, which felt very daunting. But this amazing research has shown the treatment can successfully be withdrawn and that’s fantastic.”
- Eculizumab withdrawal and monitoring in atypical haemolytic uraemic syndrome (SETS aHUS): a multicentre, open label, prospective, single arm trial. Andrew Bryant et al. The Lancet Regional Health and Europe. DOI: 10.1016/j.lanepe.2025.101392
