Longitudinal Assessment of Patient-reported Outcome Measures in Systemic Sclerosis Patients with Gastroesophageal Reflux Disease - Scleroderma Clinical Trials Consortium

Zsuzsanna H McMahan; Tracy Frech; Veronica Berrocal; David Lim; Cosimo Bruni; Marco Matucci-Cerinic; Vanessa Smith; Karin Melsens; Susanna Proudman; Jinyu Zhang; Fabian Mendoza; Melanie Woods; Dinesh Khanna

doi:10.3899/jrheum.180004

Longitudinal Assessment of Patient-reported Outcome Measures in Systemic Sclerosis Patients with Gastroesophageal Reflux Disease - Scleroderma Clinical Trials Consortium

J Rheumatol. 2019 Jan;46(1):78-84. doi: 10.3899/jrheum.180004. Epub 2018 Nov 15.

Authors

Zsuzsanna H McMahan^{1

2}, Tracy Frech^{1

2}, Veronica Berrocal^{1

2}, David Lim^{1

2}, Cosimo Bruni^{1

2}, Marco Matucci-Cerinic^{1

2}, Vanessa Smith^{1

2}, Karin Melsens^{1

2}, Susanna Proudman^{1

2}, Jinyu Zhang^{1

2}, Fabian Mendoza^{1

2}, Melanie Woods^{1

2}, Dinesh Khanna^{3

4}

Affiliations

¹ From the Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, Maryland; Division of Rheumatology, University of Utah, Salt Lake City, Utah; Department of Biostatistics, and Division of Rheumatology, University of Michigan, Ann Arbor Michigan, USA; Division of Rheumatology, University of Florence, Florence, Italy; Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University, Ghent, Belgium; Rheumatology Unit, Royal Adelaide Hospital, Discipline of Medicine, University of Adelaide, Adelaide, Australia; Department of Medicine, and Division of Rheumatology, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, USA.
² Z.H. McMahan, Assistant Professor, MD, MHS, Division of Rheumatology, Johns Hopkins University School of Medicine; T. Frech, Associate Professor, MD, MS, Division of Rheumatology, University of Utah; V. Berrocal, Associate Professor, PhD, Department of Biostatistics, University of Michigan; D. Lim, PhD student, BS, Department of Biostatistics, University of Michigan; C. Bruni, Clinical Research Fellow, MD, Division of Rheumatology, University of Florence; M. Matucci-Cerinic, Professor, MD, PhD, Division of Rheumatology, University of Florence; V. Smith, Associate Professor, MD, PhD, Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University; K. Melsens, MSc, PhD student, Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University; S. Proudman, Associate Professor, MBBS, Rheumatology Unit, Royal Adelaide Hospital, Discipline of Medicine, University of Adelaide; J. Zhang, Gastroenterology Fellow, MD, Department of Medicine, Thomas Jefferson University Hospital; F. Mendoza, Assistant Professor, MD, Division of Rheumatology, Thomas Jefferson University Hospital; M. Woods, Department of Biostatistics, University of Michigan; D. Khanna, Professor, MD, MS, Division of Rheumatology, University of Michigan. Dr. Z.H. McMahan and Dr. T. Frech contributed equally to this work.
³ From the Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, Maryland; Division of Rheumatology, University of Utah, Salt Lake City, Utah; Department of Biostatistics, and Division of Rheumatology, University of Michigan, Ann Arbor Michigan, USA; Division of Rheumatology, University of Florence, Florence, Italy; Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University, Ghent, Belgium; Rheumatology Unit, Royal Adelaide Hospital, Discipline of Medicine, University of Adelaide, Adelaide, Australia; Department of Medicine, and Division of Rheumatology, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, USA. [email protected].
⁴ Z.H. McMahan, Assistant Professor, MD, MHS, Division of Rheumatology, Johns Hopkins University School of Medicine; T. Frech, Associate Professor, MD, MS, Division of Rheumatology, University of Utah; V. Berrocal, Associate Professor, PhD, Department of Biostatistics, University of Michigan; D. Lim, PhD student, BS, Department of Biostatistics, University of Michigan; C. Bruni, Clinical Research Fellow, MD, Division of Rheumatology, University of Florence; M. Matucci-Cerinic, Professor, MD, PhD, Division of Rheumatology, University of Florence; V. Smith, Associate Professor, MD, PhD, Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University; K. Melsens, MSc, PhD student, Division of Rheumatology, Ghent University Hospital, Faculty of Internal Medicine, Ghent University; S. Proudman, Associate Professor, MBBS, Rheumatology Unit, Royal Adelaide Hospital, Discipline of Medicine, University of Adelaide; J. Zhang, Gastroenterology Fellow, MD, Department of Medicine, Thomas Jefferson University Hospital; F. Mendoza, Assistant Professor, MD, Division of Rheumatology, Thomas Jefferson University Hospital; M. Woods, Department of Biostatistics, University of Michigan; D. Khanna, Professor, MD, MS, Division of Rheumatology, University of Michigan. Dr. Z.H. McMahan and Dr. T. Frech contributed equally to this work. [email protected].

PMID: 30442827
DOI: 10.3899/jrheum.180004

Abstract

Objective: Validated gastrointestinal (GI) symptoms scales are used in clinical practice to assess patient-reported GI involvement. We sought to determine whether University of California, Los Angeles (UCLA) GI Tract Questionnaire (GIT) 2.0 Reflux scale, Patient-Reported Outcomes Measurement Information System (PROMIS) Reflux scale, and the Quality of Life in Reflux and Dyspepsia questionnaire (QOLRAD) are sensitive to identifying changes in GI symptoms following therapeutic intervention in participants with systemic sclerosis (SSc) and gastroesophageal reflux disease (GERD).

Methods: Participants with active GERD were recruited during clinical visits at 6 international SSc centers. Patient-reported outcome surveys and the GI self-reported questionnaire were completed at baseline and again at 4 weeks following a single intervention, and patients were classified as "improved" or "not improved." Effect size (ES) was calculated to assess the sensitivity to change. ES was interpreted as 0.50-0.79 as moderate effect and ≥ 0.80 as large effect.

Results: There were 116 participants with SSc and active GERD who enrolled. The average age was 53.8 years and mean disease duration was 12.0 years. The UCLA GIT 2.0 Reflux scale and PROMIS Reflux scale had a significant correlation at baseline (0.61, p < 0.0001), and both instruments correlated with the QOLRAD domains (-0.56 to -0.71). In participants who had the UCLA GIT 2.0, PROMIS Reflux scale, and QOLRAD administered over 2 timepoints (n = 57) and were classified as improved, the ES was large for the UCLA GIT 2.0 and PROMIS Reflux scale, and moderate to large across all QOLRAD domains.

Conclusion: The UCLA GIT 2.0 Reflux scale, PROMIS Reflux scale, and QOLRAD are sensitive to change and can be included in future clinical trials.

Keywords: GASTROESOPHAGEAL REFLUX DISEASE; GASTROINTESTINAL TRACT; OUTCOME ASSESSMENT; SCLERODERMA; SYSTEMIC SCLEROSIS.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adult
Aged
Aged, 80 and over
Antacids / therapeutic use*
Diet
Female
Gastroesophageal Reflux / complications
Gastroesophageal Reflux / diagnosis
Gastroesophageal Reflux / drug therapy*
Humans
Life Style
Male
Middle Aged
Patient Reported Outcome Measures
Proton Pump Inhibitors / therapeutic use*
Quality of Life
Scleroderma, Systemic / complications*
Severity of Illness Index
Surveys and Questionnaires
Treatment Outcome
Young Adult

Substances

Antacids
Proton Pump Inhibitors