A systematic review of systemic sclerosis instruments for the eular outcome measures library : An evolutionary database of validated patient-reported instruments

Background: Over time, a patient-centered evaluation of health status has become more important for systemic sclerosis (SSc), both in research and clinical setting. Patient-reported outcomes (PROs) are being increasingly used to measure various domains of disease status relevant to patients and physicians. The EULAR Outcome Measures Library (OML) is a freely available website with structured access to a growing database of validated PROs [1], but currently there are no PROs available on SSc at the EULAR OML. Objectives: To provide a comprehensive review of validated SSc-specific PROs and to critically appraise their validity. Methods: A sensitive search was developed in Medline and Embase (08/2015) to identify all validation studies, cohort studies, reviews or metaanalyses in which the objective were the development or validation of PROs evaluating organ involvement, disease activity or damage in SSc. A reviewer screened title and abstracts, selected the studies, and collected data concerning validation using ad hoc forms based on the COSMIN checklist. Results: From 13,140 articles captured, 74 met the predefined criteria. After excluding an instrument for the unavailability of an English version, the selected studies provided information on 6 SSc-specific PROs: the Scleroderma Assessment Questionnaire (SAQ), the scleroderma functional score (FS), the Raynaud's condition score (RCS), the Mouth Handicap in SSc (MHISS), the University of California Los Angeles-Scleroderma Clinical Trial Consortium Gastro-Intestinal tract (UCLA-SCTC-GIT 2.0), and the Skin Self-Assessment. The table summarizes the instruments and their measurement properties: Table 1 SSc-specific PROs Domains No. of items and range Measurement properties Reliability IC/TR/ME Validity Responsiveness Interpretability SAQ Functional status (vascular, respiratory, GIT and musculoskeletal apparatus) Items: 23Range: 0–3 ICC 0.79–0.95 Total score higher in pts with systemic involvement Vascular z=0.92–2.97; Respiratory z=1.34–2.52; GIT z=−3.14–4.03; Musculoskeletal status z=0.68–3.16 – FS Functional ability (upper limbs & muscle weakness) Items: 11Range: 0–33 Intra-observkw 0.19–0.6inter-observkw0.69–0.94 HAQ-DI r=0.90Skin score r=0.11 FS correlates with HAQ-DI 0.59, & Hand HAQ-DI 0.58 – RCS Severity and impact of Raynaud's phenomenon Item: 1Range 0–10 ICC 0.70 Disease activity, Rp measures, digital ulcers, mood/tension 67% variance ES 0.6SRM 0.64 MID 14–15 points (0–100 VAS)PASS 34 points MHISS Disability involving the mouth Items: 12Range: 0–48 ICC 0.96 HAQ r=0.33, CHFS r=0.37, mouth opening r=−0.34, MACTAR r=0.11, HADd r=0.26, HADa r=0.17 – – UCLA SCTC GIT 2.0 GIT symptoms severity Items: 34Range: 0–3 Cronbach's α>0.70, constipation (α=0.67)ICC 0.71 Total GIT score r=0.60; Upper GIT r=0.52; Lower GIT r=0.60 rho 0.05–0.48 MID/improvement: 0.07–0.36;MID/worsening: 0.06 −0.21;Floor: noneCeiling: 4% Skin self-assessment Skin thickening Items: 17Range: 0–51 ICC 0.5–0.61 Skin score r=0.435 No changes 1 yr follow-up – dcSScFloor <15%Ceiling <15%– lcSScFloor >15%Ceiling n.a. Conclusions: Six SSc-specific PROs have a minimum validation and will be included in the EULAR OML. In general, the level of validation attained could be improved. Further development in the area of disease-specific PROs in SSc is warranted. References: Castrejón I, Gossec L, Carmona L. Ann Rheum Dis. 2015;74(2):475–6