# Refining outcome measures through observational cohorts to advance trials in morphea

> **NIH NIH R01** · UT SOUTHWESTERN MEDICAL CENTER · 2020 · $210,450

## Abstract

PROJECT SUMMARY
Morphea, localized scleroderma, is an inflammatory disorder that causes thickening and hardening of affected
skin in children and adults. This produces devastating cosmetic and functional impairment. Despite an
estimated prevalence similar to type I diabetes mellitus and greater than that of systemic sclerosis, there is
only one treatment with randomized control trial evidence for efficacy in morphea, methotrexate, the use of
which is greatly limited by toxicity. New, less toxic treatment options (mycophenolate mofetil, abatacept, and
others) have shown promise in case series and observational studies, but trials are needed to examine safety
and efficacy of these promising therapies. The problem is that clinical and patient reported outcomes in
morphea are not ready for use in clinical trials. The Morphea in Adults and Children (MAC) and National
Registry for Childhood Onset Scleroderma (NRCOS) registries, led by the principal investigators, have already
begun to address this problem. Working together, we have validated the Localized Scleroderma Assessment
Tool (LoSCAT), the only clinical measure in morphea incorporating domains for activity and damage and
created using OMERACT criteria; and the pediatric Localized Scleroderma Life Quality Index (LoSQI), the only
disease specific patient reported outcome measure in morphea. Despite this progress, the sensitivity to change
and minimal clinically important difference of the LoSCAT needs to be examined before it can be used in
clinical trials and the LoSQI has not been validated in adults.
This proposal leverages the resources of the MAC and NCROS registries (the largest in the world) and the
multidisciplinary and complementary expertise of the investigators (including facilities at UTSW Medical Center
and the University of Pittsburgh) to conduct observational studies to fully validate outcomes and biomarkers for
use in clinical trials. The objectives of the proposed studies are to: 1) Determine sensitivity and minimal
clinically important difference of the LoSCAT activity component 2) Adapt the pediatric LoSQI for adult use 3)
and create a real time online dashboard to visualize and integrate clinical and patient reported outcomes
longitudinally. The conduct of these studies will not only create the outcomes and data platform necessary to
conduct well designed clinical trials in morphea, but also fully develop the multisite collaborations necessary for
trials in a rare disease.

## Key facts

- **NIH application ID:** 9882221
- **Project number:** 5R01AR073516-02
- **Recipient organization:** UT SOUTHWESTERN MEDICAL CENTER
- **Principal Investigator:** Heidi T Jacobe
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $210,450
- **Award type:** 5
- **Project period:** 2019-04-01 → 2022-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9882221

## Citation

> US National Institutes of Health, RePORTER application 9882221, Refining outcome measures through observational cohorts to advance trials in morphea (5R01AR073516-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/9882221. Licensed CC0.

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