Kidney Drug Withdrawal Offers Hope, Saves NHS Millions

Groundbreaking research indicates a pivotal shift in managing a rare, life-threatening disease.

Patients with a rare kidney condition, Atypical Haemolytic Uraemic Syndrome (aHUS), may soon be able to discontinue a vital treatment earlier than previously thought, leading to improved well-being and substantial cost savings for the UK’s National Health Service (NHS).

A Paradigm Shift in aHUS Management

New research spearheaded by Newcastle University, in collaboration with Newcastle Hospitals, suggests that most patients treated with the drug eculizumab can safely cease therapy after just six months. This development could free individuals from the burdens of regular intravenous infusions and mitigate serious infection risks associated with the medication. The findings, published in *The Lancet Regional Health Europe*, indicate that a targeted approach to this high-cost treatment could save the NHS an estimated £4.2 million per patient over their lifetime.

“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 realized in this patient population through more targeted use of this high-cost treatment.”

—Professor Neil Sherrin, Professor of Nephrology at Newcastle University

Previously, the recommendation was for patients to remain on eculizumab indefinitely unless medically advised otherwise. However, the drug carries a significantly increased risk, between 500 and 1,000 times higher, of meningococcal sepsis, a risk that is eliminated upon discontinuation.

Trial Successes and Future Directions

The study involved 28 participants aged two to 59 from across England and Scotland, all of whom had been on eculizumab for at least six months. Treatment was withdrawn for all, and remarkably, only four individuals experienced a relapse of their kidney disease. The remaining 24 patients have remained free of the condition since. This evidence supports the safety and efficacy of early withdrawal, marking a significant advancement in understanding aHUS treatment protocols.

The National Institute for Health and Care Research (NIHR), which funded the trial, highlighted the importance of integrating clinical research within the NHS. Professor Anthony Gordon, Programme Director for the NIHR Health Technology Assessment (HTA) Programme, stated that the trial’s results not only demonstrate the potential for safer, more effective treatments but also underscore the value of cost-effective care delivery.

Experts at the National Renal Complement Therapeutics Centre are continuing to closely monitor patients, aiming to answer crucial questions about the potential for re-withdrawal after a relapse and developing more accurate predictions for relapse risk following initial treatment cessation.

A Patient’s Perspective: A Renewed Quality of Life

Louise Percival, a 35-year-old diagnosed with aHUS in 2017, shared her positive experience after participating in the Newcastle University study. Previously enduring debilitating side effects such as migraines, hair loss, and breathlessness, which significantly impacted her active lifestyle, Percival found the bi-weekly infusions disruptive to her social and personal pursuits. She also experienced distress due to a fear of needles, making the cannula insertions particularly challenging.

Since stopping eculizumab under the trial’s supervision, Percival has experienced no relapses and a dramatic improvement in her overall quality of life. “All of the side effects disappeared and I feel like myself again,” she commented, expressing immense relief that what she initially believed was a lifelong commitment to treatment could indeed be successfully withdrawn.

The potential savings for the NHS are substantial, estimated at £110.4 million over five years if this targeted treatment approach is widely adopted. This advancement offers a beacon of hope for improved patient outcomes and a more sustainable healthcare system.