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Extended report
Development and validation of a patient-reported outcome instrument for skin involvement in patients with systemic sclerosis
  1. Ada Man1,
  2. Jeannette K Correa2,
  3. Jessica Ziemek3,
  4. Robert W Simms3,
  5. David T Felson3,4,
  6. Robert Lafyatis5
  1. 1 Section of Rheumatology, University of Manitoba, Winnipeg, Manitoba, Canada
  2. 2 Department of Psychology, Boston University, Boston, Massachusetts, USA
  3. 3 Section of Rheumatology, Boston University School of Medicine, Boston, Massachusetts, USA
  4. 4 NIHR Manchester Biomedical Research Unit, Manchester, UK
  5. 5 Division of Rheumatology and Clinical Immunology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA
  1. Correspondence to Dr Ada Man, Arthritis Center/Rheumatology, University of Manitoba, RR149-800 Sherbrook St, Winnipeg, Manitoba, Canada R3A 1M4; aman{at}


Objectives We developed a patient-reported outcome (PRO) instrument to assess the skin-related quality of life in patients with systemic sclerosis (SSc).

Methods Participants with SSc provided input on skin-related health effects through focus groups. We developed items for scleroderma skin PRO (SSPRO) to encompass these effects. Further consideration from cognitive interviews and an expert panel led to reduction and modification of items. A 22-item SSPRO was field tested. Psychometric analysis included test–retest reliability, internal consistency and exploratory factor analysis (EFA). Construct validity was assessed through correlation with other participant and physician-assessed measures.

Results 140 participants completed the SSPRO: mean age was 53.4 years, median disease duration was 5 years, 82.1% were female and 32.9% had diffuse cutaneous SSc. EFA supported four factors in SSPRO corresponding to hypothesised constructs: physical effects, physical limitations, emotional effects and social effects. Removal of 4/22 items resulted in acceptable goodness-of-fit statistics. Test–retest reliability (intraclass correlation coefficient=0.61–0.83) was moderate to high and internal consistency (Cronbach's α=0.89–0.96) was high. SSPRO correlated strongly with other participant-reported measures (r=0.59–0.88) suggesting construct validity, and less well with physician-assessed measures (r=0.31–0.40). SSPRO scores were significantly different for each level of participant-reported skin severity, and for limited versus diffuse cutaneous SSc.

Conclusions SSPRO has been developed with extensive patient input and demonstrates evidence for reliability and validity. It is complementary to existing measures of SSc skin involvement with emphasis on the patient's experience. Further research is needed to assess its sensitivity to change.

  • Systemic Sclerosis
  • Qualitative research
  • Outcomes research
  • Patient perspective

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  • Handling editor Tore K Kvien

  • Contributors Study concept and design: all. Acquisition of data: AM, JZ, RWS, RL. Analysis and interpretation of data: all. Statistical analysis: AM, JKC. Drafting of manuscript: AM, JKC. Critical revision of manuscript for important intellectual content: all. AM had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.

  • Funding This work was supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health under awards P50AR060780 and P60AR047785 and Boston University Medical Center CTSI: UL1-TR000157.

  • Competing interests None declared.

  • Ethics approval Boston University Institutional Review Board.

  • Provenance and peer review Not commissioned; externally peer reviewed.