Journal Article

Clinical Response Is Associated with Improvement in Health-Related Quality of Life in Patients with Persistent GERD Symptoms

Hanlon, Jennifer T, Jiang, Hancheng, Taylor, Douglas C A, Ojo, Oluwafunke, Shea, Elizabeth P, Shao, James

2018

Overview

Introduction: Responder endpoints are often used in clinical trial design to evaluate treatment efficacy, but positive responder-endpoint results are not necessarily associated with quality-of-life improvements. We evaluate the relationship between clinical responder endpoints and health-related quality of life (HRQOL) measures in patients with persistent gastroesophageal reflux disease (pGERD). Methods: Patients with pGERD - characterized by continued heartburn and regurgitation symptoms despite ongoing proton pump inhibitor (PPI) treatment-were enrolled in an 8-week, randomized, double-blinded, placebo-controlled phase 2b trial of IW-3718, an investigational gastric-retentive formulation of a bile acid sequestrant. Each patient completed a symptom assessment e-dairy (mRESQ-eD) daily, degree of relief assessments weekly, and the EQ-5D HRQOL at week 0 (baseline), 4, & 8. Heartburn and regurgitation responders were defined as having a >45% reduction in the mean daily heartburn severity or mean daily regurgitation frequency score, respectively, for >4 of 8 weeks, including >1 of the last 2 weeks. Degree of relief responders (heartburn, regurgitation, and overall GERD symptoms) were patients who reported significant or moderate relief for >4 of 8 weeks. Least-square mean changes from baseline to week 8 in EQ-5D were obtained for responders vs. non-responders using a mixed model for repeated measures (MMRM) with week (categorical), responder status, and week-by-responder status as fixed-effect terms and baseline EQ-5D as a covariate. Results: Patient demographics are shown in the Table. Responders showed better improvement in EQ-5D at week 8 compared with non-responders across all responder endpoints (p<0.05 for heartburn, regurgitation, and degree of GERD relief responder endpoints, Figure). Conclusion: Patients with persistent GERD symptoms who achieved clinical response also showed improvement in health-related quality of life as measured by the EQ-5D utility index; the heartburn, regurgitation, and GERD relief responders show significant improvement in EQ-5D results compared to non-responders. The clinical responder endpoints used in the IW-3718 phase 2b trial may yield results useful to payers as well as clinicians.

• Publisher: Wolters Kluwer Health Medical Research, Lippincott Williams & Wilkins
• Subject: Gastroesophageal reflux / Patients / Quality of life