Garth Fuller1, Roger Bolus1,2,3, Cynthia Whitman1,4, Jennifer Talley1, M Haim Erder5, Alain Joseph6, Debra G Silberg5, Brennan Spiegel7,8,9,10. 1. Cedars-Sinai Center for Outcomes Research and Education (CS-CORE), 116 N. Robertson Blvd.Suite 400, Los Angeles, CA, 90048, USA. 2. UCLA Fielding School of Public Health, Los Angeles, CA, USA. 3. , 1016 Quail Gardens Ct, Encinitas, CA, 92024, USA. 4. , 44 16th Street, Hermosa Beach, CA, 90254, USA. 5. M. H. Erder Health Economics, Inc, Livingston, NJ, USA. 6. Shire, Zählerweg 10, 6300, Zug, Switzerland. 7. Cedars-Sinai Center for Outcomes Research and Education (CS-CORE), 116 N. Robertson Blvd.Suite 400, Los Angeles, CA, 90048, USA. brennan.spiegel@cshs.org. 8. UCLA Fielding School of Public Health, Los Angeles, CA, USA. brennan.spiegel@cshs.org. 9. Department of Medicine, Cedars-Sinai Health System, Cedars-Sinai Medical Center, Los Angeles, CA, USA. brennan.spiegel@cshs.org. 10. VA Greater Los Angeles Healthcare System, Los Angeles, CA, USA. brennan.spiegel@cshs.org.
Abstract
BACKGROUND: Most patients with gastroesophageal reflux disease (GERD) experience relief following treatment with proton pump inhibitors (PPIs) (Vakil et al. in Am J Gastroenterol 101:1900-1920, 2006; Everhart and Ruhl in Gastroenterology 136:376-386, 2009). As many as 17-44% of patients, however, exhibit only partial response to therapy. Most extant GERD patient-reported outcome (PRO) instruments fail to meet development best practices as described by the FDA (Talley and Wiklund in Qual Life Res 14:21-33, 2005; Van Pinxteren et al. in Cochrane Database Syst Rev 18:CD002095, 2004; El-Serag et al. in Aliment Pharmacol Ther 32:720-737, 2010). AIM: To develop and validate a PRO instrument for clinical trials involving patients with GERD who are PPI partial responders. METHODS: We prepared a systematic literature review, held patient focus groups, convened an expert panel, and conducted cognitive interviews to establish content validity. Eligible participants took PPI therapy for at least 8 weeks, had undergone an upper endoscopy, and scored at least 8 points on the GerdQ [6]. Qualitative data guided development of 26 draft items. Items were reviewed by expert panels and debriefed with patients. The resulting 21-item instrument underwent psychometric evaluation during a Phase IIB trial. RESULTS: During the trial, confirmatory factor analysis (n = 220) resulted in a four-factor model displaying the highest goodness of fit. All domains had a high inter-item correlation (Cronbach's α > 0.8). Test-retest reliability and convergent validity were strong, with highly significant (p < 0.01) correlations between average weekly PRISM scores and severity anchors and significant (p < 0.05) correlations with anchor subscales. Cumulative distribution functions revealed significant differences between responders and non-responders. CONCLUSIONS: Analysis in a clinical trial setting demonstrated strong psychometric properties suggesting validity of PRISM. Developed in line with FDA guidance on PROs, PRISM represents an important new outcome measure for patients with GERD with a partial response to PPI therapy.
BACKGROUND: Most patients with gastroesophageal reflux disease (GERD) experience relief following treatment with proton pump inhibitors (PPIs) (Vakil et al. in Am J Gastroenterol 101:1900-1920, 2006; Everhart and Ruhl in Gastroenterology 136:376-386, 2009). As many as 17-44% of patients, however, exhibit only partial response to therapy. Most extant GERDpatient-reported outcome (PRO) instruments fail to meet development best practices as described by the FDA (Talley and Wiklund in Qual Life Res 14:21-33, 2005; Van Pinxteren et al. in Cochrane Database Syst Rev 18:CD002095, 2004; El-Serag et al. in Aliment Pharmacol Ther 32:720-737, 2010). AIM: To develop and validate a PRO instrument for clinical trials involving patients with GERD who are PPI partial responders. METHODS: We prepared a systematic literature review, held patient focus groups, convened an expert panel, and conducted cognitive interviews to establish content validity. Eligible participants took PPI therapy for at least 8 weeks, had undergone an upper endoscopy, and scored at least 8 points on the GerdQ [6]. Qualitative data guided development of 26 draft items. Items were reviewed by expert panels and debriefed with patients. The resulting 21-item instrument underwent psychometric evaluation during a Phase IIB trial. RESULTS: During the trial, confirmatory factor analysis (n = 220) resulted in a four-factor model displaying the highest goodness of fit. All domains had a high inter-item correlation (Cronbach's α > 0.8). Test-retest reliability and convergent validity were strong, with highly significant (p < 0.01) correlations between average weekly PRISM scores and severity anchors and significant (p < 0.05) correlations with anchor subscales. Cumulative distribution functions revealed significant differences between responders and non-responders. CONCLUSIONS: Analysis in a clinical trial setting demonstrated strong psychometric properties suggesting validity of PRISM. Developed in line with FDA guidance on PROs, PRISM represents an important new outcome measure for patients with GERD with a partial response to PPI therapy.
Entities:
Keywords:
Gastroesophageal reflux; Patient-reported outcomes; Proton pump inhibitors; Psychometrics; Qualitative research; US Food and Drug Administration; Validation studies
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