# MRI Risk Classification in Children and Young Adults with ADPKD

> **NIH NIH R56** · UNIVERSITY OF CHICAGO · 2022 · $100,000

## Abstract

PROJECT SUMMARY
 Autosomal dominant polycystic kidney disease (ADPKD) accounts for 10% of end stage kidney disease
(ESKD) in those <65 years of age. Through a major effort of the Consortium for Radiologic studies in Polycystic
Kidney Disease (CRISP) and others, the US FDA has approved the use of tolvaptan to limit kidney disease
progression in adult ADPKD patients, and additional clinical trials of other novel therapies are currently
underway. A key step in the development of these therapies has been the validation of height-corrected total
kidney volume (htTKV) obtained from magnetic resonance imaging (MRI) as a biomarker for identifying subjects
at high risk for progression, and htTKV is now used to as a key metric to assess an individual’s lifetime risk for
kidney disease progression though the Mayo Imaging Classification (MIC) schema.
 The development of effective therapies for children and young adults with ADPKD has the potential to limit
disease kidney disease progression at an earlier timepoint resulting in increased preservation of kidney function
and ultimately a longer life expectancy. Unfortunately, screening asymptomatic children (<18 years) with an
affected ADPKD parent is not currently recommended, so even affected young adults may not know they have
the disease. Further, children with ADPKD are generally not included in clinical trials, primarily due to the
absence of a validated biomarker for disease progression for ADPKD patients <15 years of age. Therefore,
despite the promising developments for adult ADPKD patients, these FDA-approved treatments and multiple
ongoing clinical trials are unavailable and/or not implemented for children and many young adults with ADPKD.
 Our preliminary data suggests that: 1) parent-offspring (ages 15-25 years) MICs are in close agreement,
suggesting a potential rationale for earlier screening; and 2) a combination of clinical imaging (TKV standardized
to body surface area, TKV/BSA) and rapid, quantitative MR Fingerprinting coupled with radiomics analysis can
provide the means to develop a risk stratification assessment for ADPKD children 6-14 years of age. The overall
objective of this multi-center proposal (University of Chicago and Cleveland Clinic / Case Western Reserve
University) is to improve the clinical care for pediatric and young adult ADPKD patient by: 1) defining the
association between the MIC of an ADPKD parent and their affected offspring (age 15-25 years) (Aim 1); and
2) developing an MRI-based risk classification for children <15 years of age (age 6-14 years) that can be used
to support future clinical trials in this age group (Aim 2). We will recruit 80 children and young adults with ADPKD
and a known affected parent. Clinical, demographic and genetic data will be obtained and subjects will undergo
cross sectional (Aim 1) as well longitudinal imaging assessments (Aim 2) for a total of 3 visits over the course
of 4 years. Confirmation of our preliminary findings would facilitate ...

## Key facts

- **NIH application ID:** 10673378
- **Project number:** 1R56DK133427-01
- **Recipient organization:** UNIVERSITY OF CHICAGO
- **Principal Investigator:** ARLENE B CHAPMAN
- **Activity code:** R56 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $100,000
- **Award type:** 1
- **Project period:** 2022-09-15 → 2024-09-14

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10673378

## Citation

> US National Institutes of Health, RePORTER application 10673378, MRI Risk Classification in Children and Young Adults with ADPKD (1R56DK133427-01). Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/grant/nih/10673378. Licensed CC0.

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