Alignment of Parent- and Child-Reported Outcomes and Histology in Eosinophilic Esophagitis Across Multiple CEGIR Sites

Publication date: Available online 29 May 2018
Source:Journal of Allergy and Clinical Immunology
Author(s): Seema S. Aceves, Eileen King, Margaret H. Collins, Guang-Yu Yang, Kelley E. Capocelli, J. Pablo Abonia, Dan Atkins, Peter A. Bonis, Christina L. Carpenter, Evan S. Dellon, Michael D. Eby, Gary W. Falk, Nirmala Gonsalves, Sandeep K. Gupta, Ikuo Hirano, Kendra Kocher, Jeffrey P. Krischer, John Leung, Jessi Lipscomb, Paul Menard-Katcher, Vincent A. Mukkada, Zhaoxing Pan, Jonathan M. Spergel, Qin Sun, Barry K. Wershil, Marc E. Rothenberg, Glenn T. Furuta
BackgroundPatient-reported outcome (PRO) metrics for eosinophilic esophagitis (EoE) have been developed and validated but not used in a multicenter pediatric population or systematically aligned with histology.ObjectiveWe sought to understand 1) the potential of care-giver report to predict patient self-reported symptoms and 2) the correlation of PRO domains with histology.MethodsSubjects 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) using the Pediatric Eosinophilic Esophagitis Symptom Score version 2 (PEESSv2.0), Pediatric QOL EoE module (PedsQL-EoE) and biopsies were analyzed using the EoE histology scoring system (EoEHSS).ResultsPEESSv2.0 parent and child report aligned across all domains (r=0.68-0.73, p<0.001). PedsQL-EoE reports correlated between parents and children across ages and multiple domains (r=0.48-0.79, p<0.001). There was a tight correlation between symptoms on PEESS2.0 and their impacts QOL both on self and parent report (p<0.001). 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 EoEHSS (p<0.05).ConclusionsParents of children with EoE aged 3-18 years old accurately reflected their children's disease symptoms and QOL. Self- and parent-reported symptoms correlate with proximal esophageal histology. Our data suggest that parent report in young children can function as an adequate marker for self-reported symptoms and that self-reported symptoms may reflect changes in tissue histology in the proximal esophagus. These findings should be considered during clinical trials for drug development.Clinical Implications.Parent report provides an accurate surrogate marker for self-reported symptoms and QOL in pediatric EoE. Self-reported symptoms may gauge proximal esophageal histology. This should be considered in clinical trial design.

Teaser

This study shows that parent report correlates with pediatric self-report of EoE symptoms and quality of life and that self-reported symptoms correlate with histologic changes. Parent report should be considered as a self-report surrogate for future clinical trials.


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