University of North Carolina School of Medicine Chapel Hill, NC, United States
Evan S. Dellon, MD, MPH, FACG1, Stella Karantzoulis, PhD, ABPP-CN2, Nicole Sparling, 2, Gina Eagle, MBBS, FRACP3, karol Knoop, RN, BS, CCRA3, James Nezamis, 3, Ashley Slagle, MS, PhD4, Jean Paty, PhD2 1University of North Carolina School of Medicine, Chapel Hill, NC; 2IQVIA, New York, NY; 3Ellodi Pharmaceuticals, LP, Lawrenceville, NJ; 4Aspen Consulting, LLC, Steamboat Springs, CO
Introduction: A key symptom of eosinophilic esophagitis (EoE) is dysphagia, which impacts patients’ lives and prompts eating behavioral modifications. Given the importance of this symptom, development of therapies to treat EoE should include evaluation of impact on dysphagia. The study aimed to explore the EoE patient experience and develop a patient reported outcome (PRO) instrument for dysphagia by establishing content validity and exploring outcomes in adults and adolescents for the Patient Reported Outcomes for the Symptoms of Eosinophilic esophagitis (PROSE).
Methods: Semi-structured one-on-one qualitative interviews were conducted with adults and adolescents with EoE recruited via advocacy groups and Rare Patient Voice. Patients described the symptoms and impacts of dysphagia. Spontaneous and prompted mentions were recorded, and saturation of concepts was assessed. Input was incorporated into a conceptual model. In the same interviews, patients provided feedback on the clarity, relevance, and appropriateness of the PROSE diary; feedback was used to refine the diary. The final diary was implemented electronically on a hand-held device for use in clinical trials.
Results: 26 patients were interviewed across 5 waves (n=5, 7, 3, 6, 5; age 15-50 years). Concept elicitation was conducted in the first 4 waves. Saturation was achieved by wave 2. Patients characterized their dysphagia experience as uncomfortable and/or painful “episodes”, typically lasting less than 5 minutes wherein it was difficult to swallow, as opposed to having continuous symptoms throughout the day. Coping mechanisms were described to alleviate episodes and further highlight the episodic nature of dysphagia. The most common reported impacts of dysphagia were food avoidance and social impacts (avoiding restaurants or social gatherings, bringing own food).
Patient perceptions of the PROSE were evaluated over 5 waves of cognitive debriefing, with refinements implemented after each wave. By wave 5, patients reported that all items and instructions were clear and appropriate. Patients were able to respond to the items as intended and indicated that the PROSE was a good assessment of their dysphagia experience.
Discussion: We successfully developed and established content validity of the PROSE, a self-reported electronic daily diary. A key aspect is that it allows patients to report episodes of dysphagia in real-time, as well as at end of day, in a way that is comprehensible to adults and adolescents.
Evan S. Dellon, MD, MPH, FACG1, Stella Karantzoulis, PhD, ABPP-CN2, Nicole Sparling, 2, Gina Eagle, MBBS, FRACP3, karol Knoop, RN, BS, CCRA3, James Nezamis, 3, Ashley Slagle, MS, PhD4, Jean Paty, PhD2. P2397 - Development and Content Validity of the PROSE Daily Diary to Measure Dysphagia Episodes in Adults and Adolescents With Eosinophilic Esophagitis, ACG 2021 Annual Scientific Meeting Abstracts. Las Vegas, Nevada: American College of Gastroenterology.