Prediction of short and long-term outcomes in childhood nephrotic syndrome

Abstract Introduction It is unknown whether steroid sensitivity (SS) and other putative risk factors collected at baseline can predict the disease course of idiopathic nephrotic syndrome (NS) in childhood. We determined whether demographic, clinical and family-reported factors at presentation can predict outcomes in idiopathic NS. Methods An observational cohort of 631 children aged 1 to 18 years diagnosed with idiopathic NS between 1993-2016 were followed to clinic discharge, 18 years of age, end stage kidney disease or last clinic visit. Baseline characteristics were age, sex, ethnicity, and initial SS. Of these, 287 (38%) also reported any family history of kidney disease, preceding infection, microscopic hematuria and history of asthma/allergies. The outcomes were complete remission after initial steroid course, need for a second line agent, frequently relapsing disease, and long-term remission. The discriminatory power of the models was described using the c-statistic. Results Overall, 25.7% of children had no further disease after their initial steroid course. Additionally, 31.2% develop frequently relapsing disease, yet 77.7% are disease-free at 18 years of age. Furthermore, 1% of children progress to end stage kidney disease. Logistic regression modeling using the different baseline exposures did not significantly improve the prediction of outcomes relative to the observed frequencies (maximum c-statistic 0.63, 95% CI 0.59-0.67). Addition of SS did not improve outcome prediction of long-term outcomes (c-statistic 0.63, 95% CI 0.54-0.70). Conclusions Demographic, clinical and family-reported characteristics, specifically SS, are not useful in predicting relapse rates or long-term remission in idiopathic NS. Further studies are needed to address factors that contribute to long-term health.