The rationale and design of Insight into Nephrotic Syndrome: Investigating Genes, Health and Therapeutics (INSIGHT): a prospective cohort study of childhood nephrotic syndrome.
BACKGROUND
Nephrotic syndrome is one of the most commonly diagnosed kidney diseases in childhood and its progressive forms can lead to chronic kidney disease (CKD) and/or end-stage renal disease (ESRD). There have been few longitudinal studies among a multi-ethnic cohort to determine potential risk factors influencing disease susceptibility, treatment response, and progression of nephrotic syndrome. Temporal relationships cannot be studied through cross-sectional study design. Understanding the interaction between various factors is critical to developing new strategies for treating children with kidney disease. We present the rationale and the study design of a longitudinal cohort study of children with nephrotic syndrome, the Insight into Nephrotic Syndrome: Investigating Genes, Health and Therapeutics (INSIGHT) study. The specific aims are to determine: 1) socio-demographic, environmental, and genetic factors that influence disease susceptibility; 2) rates of steroid treatment resistance and steroid treatment dependence, and identify factors that may modify treatment response; 3) clinical and genetic factors that influence disease susceptibility and progression to CKD and ESRD; and 4) the interaction between the course of illness and socio-demographic, environmental, and clinical risk factors.


METHODS
INSIGHT is a disease-based observational longitudinal cohort study of children with nephrotic syndrome. At baseline, participants complete questionnaires and provide biological specimen samples (blood, urine, and toenail clippings). Follow-up questionnaires and repeat biological specimen collections are performed annually for up to five years.


CONCLUSIONS
The proposed cohort will provide the structure to test various risk factors predicting or influencing disease susceptibility, treatment response, and progression to CKD among children with nephrotic syndrome.


BACKGROUND
ClinicalTrials.gov Identifier NCT01605266.
Division of Nephrology, The Hospital for Sick Children, and University of Toronto, Dalla Lana School of Public Health, 555 University Avenue, M5G 1X8, Toronto, ON, Canada.
Neesha
N
Hussain
J Anastasia
JA
Zello
Jovanka
J
Vasilevska-Ristovska
Tonny M
TM
Banh
Viral P
VP
Patel
Pranali
P
Patel
Christopher D
CD
Battiston
Diane
D
Hebert
Christoph P B
CP
Licht
Tino D
TD
Piscione
Rulan S
RS
Parekh
D015984:Causality; D002648:Child; D015331:Cohort Studies; D015897:Comorbidity; D005260:Female; D006801:Humans; D008297:Male; D009404:Nephrotic Syndrome / Q000175:diagnosis* / Q000453:epidemiology*; D009864:Ontario / Q000453:epidemiology; D011446:Prospective Studies; D051436:Renal Insufficiency, Chronic / Q000175:diagnosis* / Q000453:epidemiology*; D012107:Research Design; D018570:Risk Assessment; D012307:Risk Factors