Precision Medicine for Patients with Glomerular Disease – UROP Spring Symposium 2021

Precision Medicine for Patients with Glomerular Disease

Colette Cooper


Pronouns: she/her/hers

Research Mentor(s): Debbie Gipson, Professor of Pediatrics
Research Mentor School/College/Department: Pediatrics/Nephrology, Michigan Medicine
Presentation Date: Thursday, April 22, 2021
Session: Session 1 (10am-10:50am)
Breakout Room: Room 10
Presenter: 4

Event Link


Frequently relapsing/steroid dependent nephrotic syndrome (FR/SDNS) is a chronic health condition in children that is treated with long term immunosuppression therapies with variable response and significant treatment-associated toxicities. Precise, safe and effective therapies are needed. Objective: in order to address drug development needs, we undertook a project to prioritize FR/SDNS clinical trial outcomes and generate standard of care outcome estimates for use in clinical trial design. Methods: A clinician-investigator stakeholder group was polled to elicit input on FR/SDNS trial endpoint priorities. A literature review was conducted using PubMed and enriched with assessment of trial listings from Search terms included: Idiopathic Nephrotic Syndrome and Minimal Change Disease. Publications were required to be clinical trials in humans for inclusion. Data collection included population type, year of publication, age of participants, trial treatment groups, participants in each arm and the prioritized outcome relapse rate. A summary of the results by treatment arm was generated using forest plot of relapse rate point estimates and 95% confidence intervals by trial treatment arm. Results: Relapse rate was one of two prioritized FR/SDNS clinical trial endpoints. From published clinical trials, relapse rate point estimates varied by clinical trial treatment groups but were generally between 0.02 and 0.2 relapses per patient month. Conclusions: The identified relapse rates and distribution can be used in the design of FR/SDNS clinical trials for power and sample size estimations.

Authors: Colette Cooper, Maisha Pal, Hailey Desmond, Debbie Gipson
Research Method: Clinical Research

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