Hospital for Special Surgery New York, NY, United States
Disclosure information not submitted.
Jeong Min Yu1, John VanBuren2, Angela Child2, Jessica Alvey2, Lisa Mandl3, Laura Pinheiro4, Shervin Assassi5, Elana Bernstein6, Flavia Castelino7, Lorinda Chung8, Luke Evnin9, Tracy Frech10, Faye Hant11, Laura Hummers12, Dinesh Khanna13, Kimberly Lakin1, Dorota Lebiedz-Odrobina14, Yiming Luo15, Ashima Makol16, Jerry Molitor17, Duncan Moore18, Carrie Richardson19, Nora Sandorfi20, Ami Shah21, Ankoor Shah22, Victoria Shanmugam23, Brian Skaug24, Virginia Steen25, Elizabeth Volkmann26 and Jessica Gordon1, 1Hospital for Special Surgery, New York, NY, 2University of Utah, Salt Lake City, UT, 3Hospital for Special Surgery, Weill Cornell Medicine, New York, NY, 4Weill Cornell Medicine College, New York, NY, 5University of Texas McGovern Medical School at Houston, Houston, TX, 6Columbia University, New York, NY, 7Massachusetts General Hospital, Boston, MA, 8Department of Medicine, Division of Immunology and Rheumatology, Stanford University School of Medicine, Woodside, CA, 9Scleroderma Research Foundation, Brisbane, CA, 10Vanderbilt University Medical Center, Nashville, TN, 11Medical University of South Carolina, Charleston, SC, 12Johns Hopkins University, Baltimore, MD, 13University of Michigan, Ann Arbor, MI, 14University of Utah, Cottonwood Heights, UT, 15Division of Rheumatology, Department of Medicine, Columbia University Irving Medical Center, New York, NY, 16Mayo Clinic, Rochester, MN, Rochester, MN, 17University of Minnesota, Minneapolis, MN, 18Northwestern Memorial Hospital, Chicago, IL, 19Northwestern University, Riverside, IL, 20University of Pennsylvania, Philadelphia, PA, 21Department of Medicine, Division of Rheumatology, Johns Hopkins University School of Medicine, Ellicott City, MD, 22Duke University, Durham, NC, 23Victoria Shanmugam, MD, Great Falls, VA, 24Division of Rheumatology, University of Texas McGovern Medical School, Houston, TX, 25Georgetown University School of Medicine, Washington, DC, 26University of California Los Angeles, Los Angeles, CA
Background/Purpose: Skin disease is a hallmark of systemic sclerosis (SSc). The modified Rodnan skin score (mRSS) is physician performed measurement that assesses the extent and severity of skin thickness. However, it does not capture all of the clinical features of SSc skin disease. Patient-reported outcomes (PROs) can capture patients' perspective. The Scleroderma Skin Questionnaire (SSQ) is a novel PRO which assesses skin symptoms. To date, the SSQ has not been validated, and this study sought to assess the psychometric properties of the SSQ to generate evidence to support its further use.
Methods: We used data from CONQUER (Collaborative National Quality and Efficacy Registry), a multi-center, US-based registry of adults with early limited cutaneous (lc) and diffuse cutaneous (dc) SSc. The SSQ uses a 7-day recall period of 6 items (tight, painful, red, hard, itchy, rigid/stiff), which are graded on a 5-point Likert scale and averaged to obtain a score ranging 0-4 (4=worse). The second part of the questionnaire, the SSQ 6-Month, asks respondents to compare these symptoms to 6-months prior. Internal consistency of the SSQ was determined with Cronbach's α. Baseline response frequencies were used to investigate floor/ceiling effects. The associations between the SSQ and the mRSS and with legacy PROs [Scleroderma Health Assessment Questionnaire (SHAQ), Patient-Reported Outcomes Measurement Information System-29 (PROMIS-29), Patient Global (PtGA), and Physician Global (PGA)] were explored using Pearson's correlations. The SSQ 6-Month was correlated with 6-month changes in mRSS and in legacy PROs using mixed modeling with a bias-corrected confidence interval determined using cluster bootstrapping.
Results: We included 536 participants who completed the mRSS and SSQ (Table 1). The SSQ was higher (worse) in patients with dcSSc compared with lcSSc (1.5 vs 0.9; p< 0.001). The SSQ demonstrated internal consistency (α=0.90) and likely floor effect (Figure 1). At baseline, the SSQ was moderately correlated with the mRSS (r=0.56). The correlation was stronger among individuals with dcSSc (r=0.56) than lcSSc (r=0.22; all p< 0.05).At baseline, the SSQ was strongly correlated with PROMIS-29's physical (r=-0.52) and pain interference (r=0.61), and weakly with PROMIS-29's anxiety (r=0.24) and sleep disturbance (r=0.12; all p< 0.05). For the SHAQ, SSQ correlated strongly to HAQ (r=0.63) and severity score (r=0.61) and weakly to intestinal symptoms, breathing symptoms, Raynaud's attacks, and finger ulcers symptoms (all p< 0.05). When stratified for SSc subtype, baseline mRSS, and disease duration, similar trends for correlation were seen (Table 2). SSQ 6-Month showed correlation of r=0.21 (p< 0.05) with 6-month change in mRSS and poor correlation with change in legacy PROs (Table 2).
Conclusion: In the CONQUER registry, the SSQ demonstrated internal consistency and moderate correlation with mRSS. The SSQ showed moderate/strong correlations with a subset of legacy PROs. However, the SSQ 6-Month showed weak correlation with change in the mRSS, suggesting either a poor recall for a 6-month time span or a disconnect between the patient perception and the mRSS. This study provides initial characterization of SSQ for future validation.
Figure 1: SSQ Response Frequencies at Baseline Visits
J. Yu: None; J. VanBuren: None; A. Child: None; J. Alvey: None; L. Mandl: Annals of Internal Medicine, 12, Associate Editor, Regeneron Pharmaceuticals, 5, Up-to-Date, 9; L. Pinheiro: None; S. Assassi: AstraZeneca, 2, aTyr, 2, Boehringer Ingelheim, 2, 5, CSL Behring, 2, Janssen, 5, Merck, 2, Momenta, 5, TeneoFour, 2; E. Bernstein: None; F. Castelino: Boehringer-Ingelheim, 2, Genentech, 5, Prometheus, 5; L. Chung: Eicos Science, 1, 2, Eli Lilly, 1, 2, Genentech, 1, 2, IgM biosciences, 1, 2, Janssen, 1, 2, Kyverna, 1, 2, Mitsubishi Tanabe, 1, 2; L. Evnin: None; T. Frech: None; F. Hant: None; L. Hummers: AbbVie/Abbott, 1, Biotest, 2, Boehringer-Ingelheim, 1, 5, CSL Behring, 1, Cumberland Pharmaceuticals, 5, GlaxoSmithKlein(GSK), 5, Kadmon Corporation, 5, Medpace, 5, Mitsubishi Tanabe, 5, Prometheus, 5; D. Khanna: AbbVie, 12, DSMB, AstraZeneca, 2, Boehringer-Ingelheim, 2, Bristol-Myers Squibb, 2, 5, CSL Behring, 2, Genentech, 2, Horizon Therapeutics, 2, 5, Janssen, 2, 6, Pfizer, 5, Prometheus, 2; K. Lakin: None; D. Lebiedz-Odrobina: None; Y. Luo: None; A. Makol: Boehringer-Ingelheim, 1, Sanofi-Genzyme, 1; J. Molitor: None; D. Moore: None; C. Richardson: None; N. Sandorfi: None; A. Shah: Arena Pharmaceuticals, 5, Eicos Sciences, 5, Kadmon Corporation, 5, Medpace LLC, 5; A. Shah: None; V. Shanmugam: None; B. Skaug: None; V. Steen: None; E. Volkmann: Boehringer-Ingelheim, 2, 5, 6, CSL Behring, 2, GlaxoSmithKline, 2, Horizon, 5, Prometheus, 5, Roche, 2; J. Gordon: Cumberland Pharmaceuticals, 5, Prometheus Pharmaceuticals, 5.