Arthritis care & research
October 14, 2026 · via Europe PMC
Development of Patient-Reported Outcome Measures for Early Systemic Sclerosis Across Disease Subtypes.
Greene GJ, Perschon CR, Jaeger E, Lescoat A, Cella D, Khanna D.
AI summary — for doctors, generated from the abstract only
Two patient-reported outcome measures for early systemic sclerosis were developed from patient interviews to fill gaps in existing instruments.
What was studied
Concept elicitation interviews with 42 people with diffuse or limited cutaneous SSc within 7 years of onset, followed by concept prioritization, item mapping and expert reduction, within the COAST study following regulatory guidance.
Key findings
- Participants reported 52 symptoms and side effects across 16 domains, 57 physical function impacts across 8 domains, and 16 socioemotional concerns across 4 domains
- Raynaud's phenomenon was most frequent (81.0%), followed by swelling and pain (66.7% each), and fatigue was rated most bothersome among the top five (mean 7.20 on a 0-10 scale)
- Two candidate measures resulted: a 33-item symptom questionnaire and a 20-item function and mood questionnaire
Clinical relevance
The measures are intended for clinical trials in early SSc, so any benefit for routine care is indirect for now.
Who should care
Limitations
The abstract reports content development only; psychometric validation and performance in trials are not described, and the sample is small.
Patient-informed content for early SSc measures exists; validation is the next step.
This is an AI-generated summary of the published abstract, not a clinical recommendation. It may not reflect the full text or subsequent updates — verify against the original source before acting on it.
Abstract
Objective
Existing patient-reported outcome (PRO) measures for systemic sclerosis (SSc) have important limitations, including inadequate coverage of early disease and limited inclusion of diffuse cutaneous (dcSSc) and limited cutaneous (lcSSc) subtypes. This paper reports on the development of two PRO measures for early SSc in the Clinical Outcome Assessments for Systemic Sclerosis Clinical Trials (COAST) study, following regulatory guidance for clinical outcome assessments.
Methods
Concept elicitation interviews were conducted with 42 individuals with dcSSc or lcSSc within 7 years of disease onset. Data were analyzed using a constant comparative approach. The concept prioritization scheme integrated symptom frequency, bother and health-related quality of life ratings, subgroup analyses, and clinical expert input. Priority concepts were mapped to items from item libraries (PROMIS®, FACIT) and SSc-specific measures, with new items developed during measure refinement. The item pool was reduced through expert consensus.
Results
Across SSc subtypes, participants reported 52 symptoms and treatment side effects, spanning 16 domains. Raynaud's phenomenon was the most frequently reported symptom (81.0%), followed by swelling and pain (66.7% each); among the five most frequently reported symptoms, fatigue was rated most bothersome (mean=7.20, 0-10 scale). Participants also reported 57 physical function impacts across 8 domains, and 16 socioemotional concerns across 4 domains. Two candidate measures were constructed: COAST Scleroderma Symptom Questionnaire (33 items) and COAST Scleroderma Function and Mood Questionnaire (20 items).
Conclusions
The COAST measures address important gaps in SSc measurement, with content validity established through systematic patient input. The measures were designed for use in clinical trials to assess treatment benefit in early SSc.
DOI
10.1002/acr.80190
PMID
42870706
Research metadata (titles, abstracts, authors, publication dates) is sourced from Europe PMC, which aggregates content from PubMed/MEDLINE, PubMed Central, and other providers under the U.S. National Library of Medicine (NLM/NCBI). NLM does not claim copyright on abstracts; the original journal publishers or authors may hold rights to the full articles. AI-generated summaries are derived only from the abstract, never the full text, and are always labeled as AI-generated.
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