Capturing the Range of Disease Involvement in Localized Scleroderma: The Localized scleroderma Total Severity Scale (LoTSS)

Abstract

Juvenile localized scleroderma (jLS) is a chronic autoimmune disease commonly associated with poor outcome, including contractures, hemi‐atrophy, uveitis, and seizures. Despite improvements in treatment, >25% of jLS patients have functional impairment. To improve patient evaluation, our workgroup developed Localized scleroderma Total Severity Scale (LoTSS), an overall disease severity measure.MethodLoTSS was developed as a weighted measure by a consensus process involving literature review, surveys, case vignettes, and multi‐criteria decision analysis. Feasibility was assessed in larger Childhood Arthritis and Rheumatology Research Alliance groups. Construct validity with physician assessment and inter‐rater reliability was assessed using case vignettes. Additional evaluation was performed in a prospective patient cohort initiating treatment.ResultsLoTSS severity items were organized into modules that reflect jLS disease patterns, with modules for skin, extracutaneous, and craniofacial manifestations. Construct validity of LoTSS was supported by a strong positive correlation with physician global assessment (PGA) of severity and damage, and weak positive correlation with PGA‐Activity, as expected. LoTSS was responsive, with a small effect size identified. Moderate to excellent inter‐rater reliability was demonstrated. LoTSS was able to discriminate between patient subsets, with higher scores identified in those with greater disease burden and functional limitation.ConclusionWe developed a new LS measure for assessing cutaneous and extracutaneous severity, and have shown it to be reliable, valid, and responsive. LoTSS is the first measure that assesses and scores all the major extracutaneous manifestations in LS. Our findings suggest LoTSS could aid assessment and management of patients, and facilitate outcome evaluation in treatment studies.image