A new study led by experts from Newcastle University has found that eculizumab, a drug used to treat a rare kidney disease called Atypical Hemolytic Uraemic Syndrome (aHUS), effectively prevents kidney failure in 86% of patients.
The condition is a life-threatening genetic disorder caused by a defect in the immune system.
Clinically Significant
The drug had been approved for use by the NHS (National Health Service) in the United Kingdom in 2015, based on earlier clinical trials.
This latest study, published in the Blood Journal, confirms the earlier findings and emphasizes the importance of early treatment with eculizumab for aHUS patients.
Professor David Kavanagh, who led the research, said, “This study confirms the effectiveness of eculizumab in preventing kidney failure.
It further strengthens the importance of patients receiving early treatment as it’s lifesaving and significantly improves quality of life without the need for dialysis or a kidney transplant.”
The research represents the largest study of its kind, analyzing over 2,000 aHUS patients between 1995 and 2019.
Of these, 244 received eculizumab, with overwhelmingly positive results. However, 14% still required long-term dialysis.
Eculizumab is an expensive treatment, costing £328,000 per year per adult patient and is administered intravenously every two weeks.
It was recommended for NHS use by the National Institute for Health and Clinical Excellence (NICE) eight years ago.
Future Directions
The research team at Newcastle plans to focus on finding a cure for the small proportion of patients who do not respond well to eculizumab treatment.
Professor James Palmer of NHS England praised the collaborative effort behind this research. “These important findings provide renewed evidence that eculizumab is helping people live longer without kidney failure,” he said.
Dr. Aisling McMahon, executive director of research and policy at Kidney Research UK, also lauded the collaborative nature of the study, stating that it’s an excellent example of how lab research can translate into real-world clinical benefits for patients.
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The study was published in Blood Journal.
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