Original ResearchFull Report: Clinical—Alimentary TractDevelopment and Validation of a Symptom-Based Activity Index for Adults With Eosinophilic Esophagitis
Section snippets
Study Overview
The adult Eosinophilic Esophagitis Activity Index (EEsAI) study was performed in 3 phases, which are illustrated in Supplementary Figure 1. During the first phase, a comprehensive list of relevant items to be potentially incorporated into the PRO, endoscopy, histology, and blood biomarker instruments was generated. During the second phase, the prototypes of standardized instruments were evaluated in one patient group (evaluation group). Data derived from the PRO instrument were used to derive a
Patient Characteristics
A total of 153 and 120 adult EoE patients were recruited for the evaluation and validation phases, respectively. The characteristics of these patients are shown in Table 1. Age at inclusion, sex, ethnicity, and education level were comparable between the 2 groups. When compared with the patients in the evaluation group, the patients in the validation group were more likely to have EoE symptom onset more than 5 years before inclusion in the study (67.2% vs 52.9%), to experience self-reported
Discussion
Eosinophilic esophagitis is a young disease, and, to date, no validated PRO instruments reliably assessing disease activity have been approved by the regulatory authorities in the United States and Europe.
In this article, we describe the process of the development and validation of the adult EEsAI PRO instrument that assesses EoE symptom severity. We developed the EEsAI PRO instrument according to FDA guidelines.16 Patient surveys, focus groups, and semistructured interviews were used to gain
Acknowledgments
The authors would like to thank the following members of the FDA, Maryland, for their guidance to develop the PRO instrument: Office of New Drugs, including the Division of Gastroenterology and Inborn Errors Products: Andrew E. Mulberg, MD, and Robert Fiorentino, MD; Office of New Drugs, Rare Diseases: Anne R. Pariser, MD, MPH; and the Study Endpoints and Labeling Division: Elektra J. Papadopoulos MD, MPH, and Laurie B. Burke, RPh, MPH. The authors also would like to acknowledge the following
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Author names in bold designate shared co-first authors
Conflicts of interest Alain Schoepfer has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, Nestlé SA, and Novartis; he received consulting fees from Aptalis Pharma, Inc, and Dr Falk Pharma, GmbH. Alex Straumann has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, Nestlé SA, Novartis and Regeneron Pharmaceuticals, Inc; he received consulting fees from Actelion Pharmaceuticals Ltd, Bühlmann Laboratories AG, Dr Falk Pharma GmbH, Genentech, Inc, Glaxo Smith Kline, plc, Meritage Pharma, Inc, Merck & Co, Inc, Nestle SA, Novartis AG, Oxagen, Ltd, Pfizer, Inc, Receptos, Inc, Regeneron Pharmaceuticals, Inc; he also received speaker fees from Takeda Pharmaceutical Company, Ltd; Radoslaw Panczak has received consulting fees from Aptalis Pharma, Inc; Claudia Kuehni has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, and Nestlé SA; Yvonne Romero has collaborated on projects supported by Aptalis Pharma, Inc, and Meritage Pharma, Inc, and has received royalties for commercial use of the MDQ-30; Ikuo Hirano has received research grants from Meritage Pharma, Inc, and consulting fees from Aptalis Pharma, Inc, Meritage Pharma, Inc, and Receptos, Inc; Jeffrey Alexander has received consulting fees from Meritage Pharma, Inc, and Aptalis Pharma, Inc, and research grant from Merck & Co, Inc; he has received royalties for commercial use of the MDQ-30, and also has a financial interest in Meritage Pharma, Inc; Glenn Furuta has received consulting fees from Pfizer, Inc, Meritage Pharma, Inc, Knopp, and Biosciences, LLC, and has received royalties from UpToDate, Inc, and is also a founder of EnteroTrack, LLC; Evan Dellon has received research grants from AstraZeneca, AG, and Meritage Pharma, Inc, he has received consulting fees from Aptalis Pharma, Inc, Novartis, Receptos, Inc, and Regeneron Pharmaceuticals, Inc; John Leung has received research grants from Meritage Pharma, Inc; Margaret Collins has received consulting fees from Aptalis Pharma, Inc, Biogen Idec, Meritage Pharma, Inc, Novartis, and Receptos, Inc; Sandeep Gupta has received consulting fees from QOL, Receptos, Inc, and Meritage Pharma, Inc, and speaker fees from Abbott Laboratories and Nestlé SA. Seema Aceves is a co-inventor of oral viscous budesonide (patent held by University of California, San Diego), and also has received royalties for oral viscous budesonide from Meritage Pharma, Inc, owns stocks in Meritage Pharma, Inc, and has received consulting fees from Receptos, Inc, and Regeneron Pharmaceuticals, Inc; Felicity Enders receives royalties for commercial use of the MDQ-30; Marcel Zwahlen has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, and Nestlé SA; and Ekaterina Safroneeva has received consulting fees from Aptalis Pharma, Inc, and Novartis. The remaining authors disclose no conflicts.
Funding Supported by a grant from the Swiss National Science Foundation (32003B_135665/1), AstraZeneca AG, Aptalis, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc (MEE114518), Nestlé SA, Receptos, Inc, and The International Gastrointestinal Eosinophil ResearcherS (A.M.S., A.S., C.E.K., and M.Z.). Also supported by a grant from the National Institute of Health (1K24DK100303 to G.T.F.).
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Authors share co-first authorship.