Evaluation of Validated Patient Reported Outcome Measures to Assess Sensitivity to Change in Patients with Systemic Sclerosis and Gastroesophageal Reflux Disease —a Scleroderma Clinical Trials Consortium Collaborative Project

Zsuzsanna McMahan¹, Tracy M. Frech², David Lim³, Veronica J. Berrocal⁴, Cosimo Bruni⁵, Marco Matucci-Cerinic⁶, Vanessa Smith⁷, Karin Melsons⁸, Susanna Proudman⁹, Jinyu Zhang¹⁰, Fabian A Mendoza¹¹, Melanie Woods³ and Dinesh Khanna³, ¹Department of Internal Medicine, Johns Hopkins University, Baltimore, MD, ²Division of Rheumatology, University of Utah, Salt Lake City, UT, ³University of Michigan, Ann Arbor, MI, ⁴Div of Rheumatology, University of Michigan, Ann Arbor, MI, ⁵Department of Experimental and Clinical Medicine, Division of Rheumatology, University of Florence, Firenze, Italy, ⁶Dept of Medicine/Div of Rheum, University of Florence, Firenze, Italy, ⁷Faculty of Internal Medicine, Ghent University, Ghent, Belgium, ⁸Ghent University, Gent, Belgium, ⁹Discipline of Medicine, University of Adelaide, Adelaide, Australia, ¹⁰Thomas Jefferson University, Philadelphia, PA, ¹¹Jefferson Institute of Molecular Medicine and Scleroderma Center, Rheumatology Division, Department of Medicine, Thomas Jefferson University, Philadelphia, PA

Meeting: 2017 ACR/ARHP Annual Meeting

Date of first publication: September 18, 2017

Keywords: gastrointestinal complications, longitudinal studies, patient outcomes, scleroderma and systemic sclerosis

Session Information

Date: Monday, November 6, 2017

Title: Patient Outcomes, Preferences, and Attitudes Poster II

Session Type: ACR Poster Session B

Session Time: 9:00AM-11:00AM

Background/Purpose: The UCLA SCTC GIT 2.0 and NIH PROMIS GI Symptoms Scales are validated in scleroderma to assess patient-reported gastrointestinal (GI) involvement. We sought to determine whether longitudinally administered reflux-specific domains of the UCLA GIT 2.0 and PROMIS are effective in capturing change in GI symptoms following therapeutic intervention in an international cohort of scleroderma patients with gastroesophageal reflux disease (GERD).

Methods: Six scleroderma centers participated in this longitudinal observational cohort study. Patients with active GERD, defined as symptoms for at least 3 of the last 7 days, were recruited during routine clinical visits. Patient-reported outcomes (PROs) were captured at baseline and again at 4 weeks follow-up. Effect size and standardized response mean (SRM) were calculated to assess the sensitivity to change and a range of 0.20-0.49 was interpreted as small magnitude, 0.50-0.79 as moderate magnitude, and >=0.80 as large magnitude.

Results: 115 subjects with systemic sclerosis and active GERD were recruited. The average age was 53.7 ± 13.3 years, with a mean disease duration of 12.7 years. Patients were more likely to be female (81%), and there was a similar distribution of patients with diffuse and limited cutaneous disease (43% vs. 48%, respectively). Mean body mass index was 25.9 ± 6.2. The mean UCA GIT 2.0 Reflux score was 0.98 ± 0.63 (N=112) and mean PROMIS score was 53.5 ± 8.1 (0.3 SD above the US population; N=64). The UCLA GIT 2.0 and PROMIS had a significant correlation coefficient at baseline (-0.60, p=0.000000219). In patients where both the UCLA GIT 2.0 and PROMIS were available (N=57), the changes were associated with a small effect size and SRM during 4-week follow-up (Table 1). For subjects who reported improvement, the effect size and SRM were moderate (-0.62 and -0.54) for UCLA GIT 2.0, and large (-1.03 and -1.02) for NIH PROMIS.

Table 1. Effect size and standardized response means across patient groups
	Whole Group	Whole Group	Improved group	Improved group
Patient-Reported Outcome Scales	Effect size	SRM	Effect size*	SRM*
UCLA SCTC GIT 2.0	0.25	0.22	-0.62	-0.54
NIH PROMIS	0.28	0.33	-1.03	-1.02

Conclusion: In this unique large international cohort, both the UCLA GIT 2.0 and PROMIS are sensitive to change. UCLA GIT 2.0 and PROMIS have a large correlation for the assessment of GERD in systemic sclerosis. Although the change was of small magnitude, statistical correlation was demonstrated.

Disclosure: Z. McMahan, None; T. M. Frech, None; D. Lim, None; V. J. Berrocal, None; C. Bruni, None; M. Matucci-Cerinic, None; V. Smith, None; K. Melsons, None; S. Proudman, None; J. Zhang, None; F. A. Mendoza, None; M. Woods, None; D. Khanna, Actelion, Bayer, BoehringerIngelheim, Chemomab, Corbus, Covis, Cytori,Eicos, EMD Serono, Genentech/Roche, Gilead, GSK, Sanofi-Aventis,UCB Pharma, 5,NIH/NIAMS, NIH/NIAID,Bayer, BMS, Genentech/Roche, Pfizer, 2,Eicos, 4.

To cite this abstract in AMA style:

McMahan Z, Frech TM, Lim D, Berrocal VJ, Bruni C, Matucci-Cerinic M, Smith V, Melsons K, Proudman S, Zhang J, Mendoza FA, Woods M, Khanna D. Evaluation of Validated Patient Reported Outcome Measures to Assess Sensitivity to Change in Patients with Systemic Sclerosis and Gastroesophageal Reflux Disease —a Scleroderma Clinical Trials Consortium Collaborative Project [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). https://acrabstracts.org/abstract/evaluation-of-validated-patient-reported-outcome-measures-to-assess-sensitivity-to-change-in-patients-with-systemic-sclerosis-and-gastroesophageal-reflux-disease-a-scleroderma/. Accessed .

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