Pharmacy Daily | Rare kidney disease trial

A TRIAL investigating how children are treated for a rare kidney disease called nephrotic syndrome is currently underway.

The OPEN (Optimising Prednisolone dosE in childhood Nephrotic syndrome) trial is being led by a team at Flinders University and backed by grant funding of almost $2 million from the Australian Government's Medical Research Future Fund.

Nephrotic syndrome is a potentially life-threatening kidney condition that affects one in 50,000 children a year.

The syndrome has been treated with the same medication since the 1960s - oral prednisolone, a high-dose steroid treatment.

Unfortunately, it often comes with significant side effects like weight gain, behavioural changes, infections and impaired growth.

Relapses are also common, leading to patients then requiring multiple doses of steroids, with gradually increasing side-effects.

"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," said trial chief investigator Prof Jonathan Craig, kidney researcher and clinician and Vice President and Executive Dean of Flinders University's College of Medicine and Public Health.

"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," Craig explained.

"This approach has already shown promise which demonstrates we could potentially halve the cumulative steroid dose and alleviate the associated side effects." JHM