Seema S Aceves1, Eileen King2, Margaret H Collins3, Guang-Yu Yang4, Kelley E Capocelli5, J Pablo Abonia6, Dan Atkins7, Peter A Bonis8, Christina L Carpenter9, Evan S Dellon10, Michael D Eby6, Gary W Falk11, Nirmala Gonsalves12, Sandeep K Gupta13, Ikuo Hirano12, Kendra Kocher14, Jeffrey P Krischer9, John Leung8, Jessi Lipscomb2, Paul Menard-Katcher14, Vincent A Mukkada15, Zhaoxing Pan16, Jonathan M Spergel17, Qin Sun2, Barry K Wershil18, Marc E Rothenberg19, Glenn T Furuta14. 1. Division of Allergy Immunology, University of California, San Diego, Rady Children's Hospital, San Diego, Calif. 2. Division of Biostatistics and Epidemiology, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio. 3. Division of Pathology, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio. 4. Division of Pathology, Northwestern University, Feinberg School of Medicine, Chicago, Ill. 5. Division of Pathology, Children's Hospital Colorado, Aurora, Colo. 6. Division of Allergy and Immunology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio. 7. Section of Allergy, Immunology, Children's Hospital Colorado, Aurora, Colo. 8. Division of Gastroenterology, Tufts Medical Center, Boston, Mass. 9. Health Informatics Institute, Departments of Pediatrics and Medicine, Morsani College of Medicine, University of South Florida, Tampa, Fla. 10. Department of Gastroenterology and Hepatology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC. 11. Division of Gastroenterology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pa. 12. Division of Gastroenterology & Hepatology, Northwestern University, Feinberg School of Medicine, Chicago, Ill. 13. Department of Pediatric Gastroenterology, Hepatology and Nutrition, University of Illinois College of Medicine, Chicago, Ill. 14. Gastrointestinal Eosinophilic Diseases Program, Section of Pediatric Gastroenterology, Hepatology and Nutrition, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colo. 15. Division of Gastroenterology, Hepatology, and Nutrition, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio. 16. Pediatric Gastroenterology, Pediatric Allergy and Immunology, Children's Hospital of Colorado, Aurora, Colo. 17. Department of Allergy and Immunology, Children's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, Pa. 18. Division of Gastroenterology, Hepatology, and Nutrition, Ann & Robert Lurie Children's Hospital of Chicago, Chicago, Ill. 19. Division of Allergy and Immunology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio. Electronic address: Marc.Rothenberg@cchmc.org.
Abstract
BACKGROUND: Patient-reported outcome metrics for eosinophilic esophagitis (EoE) have been developed and validated but not used in a multicenter pediatric population or systematically aligned with histology. OBJECTIVE: We sought to understand (1) the potential of caregiver report to predict patient self-reported symptoms and (2) the correlation of patient-reported outcome domains with histology. METHODS: Patients with EoE (n = 310) and their parents participating in the Consortium of Gastrointestinal Eosinophilic Disease Researchers (CEGIR) observational clinical trial were queried for baseline patient symptoms and quality of life (QOL) by using the Pediatric Eosinophilic Esophagitis Symptom Score, version 2 (PEESSv2.0), and the Pediatric QOL EoE module (PedsQL-EoE), and biopsy specimens were analyzed by using the EoE Histology Scoring System. RESULTS: PEESSv2.0 parental and child reports aligned across all domains (r = 0.68-0.73, P < .001). PedsQL-EoE reports correlated between parents and children across ages and multiple domains (r = 0.48-0.79, P < .001). There was a tight correlation between symptoms on PEESSv2.0 and their effects on QOL both on self-report and parental report (P < .001). Self-reported symptoms on PEESSv2.0 (positively) and PedsQL-EoE (inversely) showed a weak correlation with proximal, but not distal, peak eosinophil counts and features and architectural tissue changes on the EoE Histology Scoring System (P < .05). CONCLUSIONS: Parents of children with EoE aged 3 to 18 years accurately reflected their children's disease symptoms and QOL. Self- and parent-reported symptoms correlate with proximal esophageal histology. Our data suggest that parental report in young children can function as an adequate marker for self-reported symptoms and that self-reported symptoms can reflect changes in tissue histology in the proximal esophagus. These findings should be considered during clinical trials for drug development.
BACKGROUND:Patient-reported outcome metrics for eosinophilic esophagitis (EoE) have been developed and validated but not used in a multicenter pediatric population or systematically aligned with histology. OBJECTIVE: We sought to understand (1) the potential of caregiver report to predict patient self-reported symptoms and (2) the correlation of patient-reported outcome domains with histology. METHODS:Patients with EoE (n = 310) and their parents participating in the Consortium of Gastrointestinal Eosinophilic Disease Researchers (CEGIR) observational clinical trial were queried for baseline patient symptoms and quality of life (QOL) by using the Pediatric Eosinophilic Esophagitis Symptom Score, version 2 (PEESSv2.0), and the Pediatric QOL EoE module (PedsQL-EoE), and biopsy specimens were analyzed by using the EoE Histology Scoring System. RESULTS: PEESSv2.0 parental and child reports aligned across all domains (r = 0.68-0.73, P < .001). PedsQL-EoE reports correlated between parents and children across ages and multiple domains (r = 0.48-0.79, P < .001). There was a tight correlation between symptoms on PEESSv2.0 and their effects on QOL both on self-report and parental report (P < .001). Self-reported symptoms on PEESSv2.0 (positively) and PedsQL-EoE (inversely) showed a weak correlation with proximal, but not distal, peak eosinophil counts and features and architectural tissue changes on the EoE Histology Scoring System (P < .05). CONCLUSIONS: Parents of children with EoE aged 3 to 18 years accurately reflected their children's disease symptoms and QOL. Self- and parent-reported symptoms correlate with proximal esophageal histology. Our data suggest that parental report in young children can function as an adequate marker for self-reported symptoms and that self-reported symptoms can reflect changes in tissue histology in the proximal esophagus. These findings should be considered during clinical trials for drug development.
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