A researcher at the University of Calgary is leading a national study aimed at identifying best practices in treating nephrotic syndrome, a rare childhood disorder that causes your body to excrete too much protein in the urine. There are almost 100 children affected in southern Alberta alone. It’s not known what causes the illness and there is no cure.
“It’s a challenge to generate high-quality evidence about rare illnesses like childhood nephrotic syndrome,” says Dr. Susan Samuel, an Alberta Health Services pediatric nephrologist and researcher with both the Alberta Children’s Hospital Research Institute and the O'Brien Institute for Public Health at the University of Calgary's Cumming School of Medicine.
“There are very few research studies that involve significant numbers of children, meaning we don’t know with high level of certainty what the most effective course of treatment is,” says Samuel.
In the five-year-project, 13 children’s hospitals across Canada will pool information on their patients’ responses to treatment; 140 patients are already enrolled. When it is complete, it’s expected that the patient registry and data repository will have information on more than 500 cases.
Rare disorder often misdiagnosed; study hopes to lead to more precise prognostics
Children with this rare disorder are often misdiagnosed with allergies at first because they experience puffiness and food intolerance. Those affected often have multiple relapses of the illness over several decades. Steroids have been the standard treatment since the 1950s, but there is wide variation on the total dose and duration of therapy for relapses.
Variations in the length of treatment range from six weeks to six months. Many children eventually outgrow the condition, but others stay in a cycle of relapse and remission and must undergo a course of steroid treatment every few months. Some even progress to kidney failure.
“Steroid treatment can have negative side-effects, such as obesity, slowed growth, high blood pressure, cataracts, poor bone health, and behavioural issues,” Samuel says.
“One potential benefit of the study would be to determine the minimal amount of steroids that can be prescribed while still remaining effective in preventing relapses of disease.”
Another hope is that the findings could lead to the development of precise prognostic information and targeted therapies for individual patients.
Experts at national symposium give research insights to patients and parents
The Alberta Children’s Hospital recently hosted a national symposium on nephrotic syndrome, bringing together researchers and affected families from across the country. During the symposium, the pediatric nephrology clinic staff hosted a patient and parent forum. During the forum, a panel of experts answered patients’ and families’ questions regarding the latest advances in nephrotic syndrome research and how to best care for children with this disease.
The research study is funded by the Canadian Institutes of Health Research and the Kidney Foundation of Canada.
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