Desing and method of Korean pEdiatric cohort studY for improving outcome in NephrOTic syndrome (KEYNOTE): a nationwide prospective cohort

초록

Aims/Purpose: The childhood nephrotic syndrome (NS) exhibits variable courses and prognoses, with treatment approaches differing among clinicians. Frequent relapses can lead to complications from both medications and the disease itself, significantly reducing the quality of life for patients and their families. Moreover, despite continuous treatments, some patients progress to end-stage kidney disease, yet no biomarkers are currently available to predict treatment response for NS. Previous studies have shown that clinical manifestations and medication response in NS vary across races and regions. Therefore, this cohort study aims to comprehend the characteristics of Korean pediatric NS patients. Methods: Children with nephrotic syndrome will be recruited from all 28 pediatric nephrology centers across Korea. The eligible participants will undergo evaluation for clinical data including physical examination, laboratory findings, medications, and pathologic or genetic findings at diagnosis, as well as 1/3/6 months after diagnosis, and annually thereafter. Samples of buffy coat, serum, urine, and kidney tissue will be stored for further exploration of NS biomarkers. Additionally, information on steroid response, number of relapses, cumulative dosage of medications (steroids, cyclosporine, tacrolimus, and cyclophosphamide), and drug-related complications will be collected. Events such as infection, peritonitis, fracture, Stevens-Johnsons syndrome, thromboembolism, acute kidney injury, death, renal event (including doubling of creatinine, 50% decline in glomerular filtration rate, or initiation of renal replacement therapy), and cardiovascular event will also be collected. Results: The data obtained from this cohort will be analyzed to determine the incidence, and risk factors for relapse, medication response, complications, and progression to chronic kidney disease. Conclusion: As the first nationwide cohort for pediatric NS in Korea, the KEYNOTE will contribute to a comprehensive understanding of the clinical manifestations, as well as the short-term and long-term complications, prognosis, and risk factors associated with NS.