Transforming treatment for rare childhood kidney condition

A nationwide trial led by Flinders University will seek to transform how children are treated for nephrotic syndrome, a rare and potentially life-threatening kidney condition affecting 1 in 50,000 children annually.

Characterised by the leakage of protein into the urine, leading to severe swelling, blood clotting, and susceptibility to infections, nephrotic syndrome is currently treated with oral prednisolone, a high-dose steroid treatment.

“Corticosteroid therapy has been the gold standard of care for nephrotic syndrome since the 1960s, and while this does save lives, it comes with significant side effects including weight gain, behaviour changes, infections and impaired growth,” says trial chief investigator Professor Jonathan Craig, kidney researcher and clinician and Vice President and Executive Dean of Flinders University’s College of Medicine and Public Health.

“Additionally, relapses are also very common, with children then requiring multiple doses of steroids. These doses essentially begin to accumulate overtime, further exacerbating these side-effects. While a cure is not possible, there is an urgent need to ensure we can optimise steroid dosages to effectively manage the disease, while minimising the burden to children and their families.”

Professor Jonathan Craig, Vice President and Executive Dean of the College of Medicine and Public Health at Flinders University.

Backed by recently announced grant funding of almost $2 million from the Australian Government’s Medical Research Future Fund, the clinical trial aims to investigate the best dosage for relapses of nephrotic syndrome.

“Recent clinical trials have confirmed what we give to children the first time they are diagnosed is the optimal dose, however what we don’t know yet is if there is the possibility of treating relapses with half the amount of steroids we currently use,” says Professor Craig.

“Using a randomised trial, we will compare the efficacy of a lower intensity corticosteroid treatment to the current standard with the hope this still leads to remission, and maintains remission.

“This approach has already shown promise in observational and small trial data, which demonstrates we could potentially halve the cumulative steroid dose and alleviate the associated side effects.”

The OPEN (Optimising Prednisolone dosE in childhood Nephrotic syndrome) trial is supported by all the paediatric nephrology units in Australia, who provide specialist care to children with NS, alongside several international collaborators.

The trial will also incorporate economic evaluations and patient-reported outcomes, co-designed with patient partners, to provide a comprehensive assessment of the treatment’s impact.

“The results of the OPEN trial will answer a critical and unanswered question and has the potential to transform care and outcomes for children with this rare and neglected disease worldwide,” says Professor Craig.

The project ‘A randomised trial of intensive vs less intensive corticosteroids for children with nephrotic syndrome (OPEN trial)’ has been awarded $1,959,838.95 by the Medical Research Future Fund – Clinical Trials Activity Initiative – 2022 Clinical Trials Activity Grant Opportunity – Stream 1. Project partners include Perth Children’s Hospital, University of Sydney, University of Queensland, Alberta Children’s Hospital, The Royal Children’s Hospital Melbourne and Adelaide’s Women’s and Children’s Hospital.

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