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.

Public Health Relevance

Morphea, localized scleroderma, causes thickening and hardening of the skin producing devastating functional and cosmetic disability in children and adults. There are few therapeutic options for morphea, but recent studies suggest new treatments are ready for evaluation in clinical trials. Before clinical trials can be conducted, however, fully validated clinical and patient reported outcomes are needed. This study utilizes a prospective longitudinal study to complete the validation of clinical and patient reported outcomes in morphea as well as creating a real time dashboard for integration of the results of these studies, setting the stage for the investigators to conduct clinical trials.

National Institute of Health (NIH)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Research Project (R01)
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Arthritis and Musculoskeletal and Skin Diseases Clinical Trials Review Committee (AMSC)
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Park, Heiyoung
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University of Texas Sw Medical Center Dallas
Schools of Medicine
United States
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