Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire

Abstract

ObjectivesPatient-reported outcome measures (PROMs) are important for clinical practice and research. Given the high unmet need, our aim was to develop a comprehensive PROM for systemic sclerosis (SSc), jointly with patient experts.MethodsThis European Alliance of Associations for Rheumatology (EULAR)-endorsed project involved 11 European SSc centres. Relevant health dimensions were chosen and prioritised by patients. The resulting Systemic Sclerosis Impact of Disease (ScleroID) questionnaire was subsequently weighted and validated by Outcome Measures in Rheumatology criteria in an observational cohort study, cross-sectionally and longitudinally. As comparators, SSc-Health Assessment Questionnaire (HAQ), EuroQol Five Dimensional (EQ-5D), Short Form-36 (SF-36) were included.ResultsInitially, 17 health dimensions were selected and prioritised. The top 10 health dimensions were selected for the ScleroID questionnaire. Importantly, Raynaud’s phenomenon, impaired hand function, pain and fatigue had the highest patient-reported disease impact. The validation cohort study included 472 patients with a baseline visit, from which 109 had a test–retest reliability visit and 113 had a follow-up visit (85% female, 38% diffuse SSc, mean age 58 years, mean disease duration 9 years). The total ScleroID score showed strong Pearson correlation coefficients with comparators (SSc-HAQ, 0.73; Patient’s global assessment, Visual Analogue Scale 0.77; HAQ-Disability Index, 0.62; SF-36 physical score, −0.62; each p<0.001). The internal consistency was strong: Cronbach’s alpha was 0.87, similar to SSc-HAQ (0.88) and higher than EQ-5D (0.77). The ScleroID had excellent reliability and good sensitivity to change, superior to all comparators (intraclass correlation coefficient 0.84; standardised response mean 0.57).ConclusionsWe have developed and validated the EULAR ScleroID, which is a novel, brief, disease-specific, patient-derived, disease impact PROM, suitable for research and clinical use in SSc.

Systemic sclerosis: one year in review 2022

“…The validation study showed a better sensitivity to change over a mean follow-up of 12.2 months than comparators such as Short Form-36 (SF-36), EuroQol Five Dimensional (EQ-5D), and the SSc-Health Assessment Questionnaire (HAQ). The ScleroID questionnaire proved easy to apply, had high internal consistency, and showed good correlation with the patient global assessment (109).…”

“…• The ScleroID questionnaire is a new, SSc-specific, and patient-derived PRO measure which has proven superior to comparators and is thus suitable for clinical use (109).…”

Efficacy and Safety of Botulinum Toxin in Adults with Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Multicenter, Randomized, Double‐Blind, Placebo‐Controlled Study

“…Supported by the Programme Hospitalier de Recherche Clinique National (Ministry of Health grant PHRC P170904J). and pain are the highest patient-reported disease impacts of SSc-RP (3); however, treatment is challenging and considered an unmet clinical need by patients (3)(4)(5)(6). Patients are commonly told to avoid RP triggers, such as cold exposure, tobacco, and stress.…”

“…Thus, SSc‐associated RP (SSc‐RP) may appear several years before systemic manifestations and can be further complicated by permanent chronic digital ischemia, including ulceration and necrosis (1,2). Impaired hand function and pain are the highest patient‐reported disease impacts of SSc‐RP (3); however, treatment is challenging and considered an unmet clinical need by patients (3–6). Patients are commonly told to avoid RP triggers, such as cold exposure, tobacco, and stress.…”

Composite measures to assess disease damage, disease severity and treatment response in systemic sclerosis clinical trials

“…Therefore, the limitations of the face and content validity of the MSS require an appraisal of how best to assess overall disease burden in SSc (Table 1). This question of assessing overall disease burden has recently been addressed by a team of European Alliance of Associations for Rheumatology (EULAR) researchers who have developed the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire [22 ▪▪ ].…”