Steroid Sensitive Nephrotic Syndrome in Children

Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in
children and corticosteroid therapy is its most effective treatment. The main objective of
our prospective, open-label, observational clinical cohort study is to test the feasibility
of a shorter duration of corticosteroid therapy in patients who show a quicker treatment
response. We hypothesize that the clinical outcomes in children with time to remission of ≤10
days and treated with only 8 weeks of corticosteroid therapy will not be significantly
different as compared to those with time to remission of >10 days and treated with ≥12 weeks
of standard corticosteroid therapy. Our specific aims are as follows: First, we will evaluate
the time to first relapse after 8-week corticosteroid therapy in quick responders in
comparison to the standard treatment of ≥12 weeks in slow responders. Second, we will assess
the frequency of relapses during one year follow-up after completion of 8-week corticosteroid
therapy in quick responders in comparison to the standard treatment of ≥12 weeks in slow
responders. To complete the study successfully during the funding period of two years and to
increase the generalizability of its results, the study will recruit 66 patients at six study
participating sites in five countries, including U.S., India, China, Egypt, and Qatar. The
sites have been carefully selected on the basis of their reputation, patient volume, research
experience, and PI's personal rapport with the site investigators. The proposed study is
innovative because it seeks a paradigm shift from 'one-size-fits-all' to an entirely new
concept of individualized treatment duration based on "time to remission" with initial
corticosteroid therapy. The proposed study is the first precision medicine initiative in the
management of INS. The project is significant because of the potential to improve public
health by decreasing the side effects of prolonged corticosteroid administration in about
half of the patients diagnosed with INS. Our long-term objective is to develop additional
novel therapeutic strategies to optimize the use of corticosteroids in the management of
initial episode and relapses in children with INS.

Inclusion Criteria:

- Age 1 to <19 years

- Newly diagnosed INS

- Patient in remission with steroids

- Written informed consent/Assent for the study OR as required by the local IRB

Exclusion Criteria:

- Age < 1 year or ≥ 19 years

- Uncertainty about patient/parent adherence.

- Abnormal serum creatinine for patient age

- Steroid resistant nephrotic syndrome

- Any co-morbid condition that might require modification in treatment with steroids.