TY - JOUR
T1 - Alignment of parent- and child-reported outcomes and histology in eosinophilic esophagitis across multiple CEGIR sites
AU - Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR)
AU - Aceves, Seema S.
AU - King, Eileen
AU - Collins, Margaret H.
AU - Yang, Guang-Yu
AU - Capocelli, Kelley E.
AU - Abonia, J. Pablo
AU - Atkins, Dan
AU - Bonis, Peter A.
AU - Carpenter, Christina L.
AU - Dellon, Evan S.
AU - Eby, Michael D.
AU - Falk, Gary W.
AU - Gonsalves, Nirmala Prabu
AU - Gupta, Sandeep K.
AU - Hirano, Ikuo
AU - Kocher, Kendra
AU - Krischer, Jeffrey P.
AU - Leung, John
AU - Lipscomb, Jessi
AU - Menard-Katcher, Paul
AU - Mukkada, Vincent A.
AU - Pan, Zhaoxing
AU - Spergel, Jonathan M.
AU - Sun, Qin
AU - Wershil, Barry K
AU - Rothenberg, Marc E.
AU - Furuta, Glenn T.
AU - Arrington, Ashley
AU - Bailey, Jeanie
AU - Besse, John
AU - Book, Wendy M.
AU - Burke, Deirdre
AU - Covington, Jacquelyn
AU - DeMarschall, Maureen
AU - Dohil, Ranjan
AU - Dubner, Allison
AU - Foote, Heather
AU - Guan, Shaobo
AU - Hurnton, Alicia
AU - Kodroff, Ellyn
AU - Kuhl, Jonathan
AU - Kyle, Shay
AU - Lewis, Megan
AU - Mack, Denise
AU - McGee, Sarah
AU - Mingler, Melissa
AU - Moist, Susan
AU - Muir, Amanda
AU - Poppendeck, Heidi
AU - Wechsler, Joshua Brian
N1 - Funding Information:
We thank Amanda Rudman-Spergel for guidance and input, Shawna Hottinger for editorial assistance, colleagues and clinical support staff in CEGIR for procuring biopsy specimens and clinical data, and families for participating in the trial. This study was supported by CEGIR (U54 AI117804), which is part of the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research (ORDR), NCATS, and is co-funded by the NIAID, NIDDK, and NCATS. CEGIR is also supported by patient advocacy groups including the American Partnership for Eosinophilic Disorders (APFED), Campaign Urging Research for Eosinophilic Disease (CURED), and Eosinophilic Family Coalition (EFC).
Publisher Copyright:
© 2018 American Academy of Allergy, Asthma & Immunology
PY - 2018/7
Y1 - 2018/7
N2 - 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.
AB - 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.
KW - Consortium of Eosinophilic Gastrointestinal Disease Researchers
KW - Eosinophil
KW - eosinophilic esophagitis
KW - eosinophilic oesophagitis
KW - patient-reported outcomes
KW - pediatric QOL EoE module
KW - pediatric eosinophilic esophagitis symptom score
KW - quality of life
KW - symptoms
KW - version 2
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UR - http://www.scopus.com/inward/citedby.url?scp=85049074301&partnerID=8YFLogxK
U2 - 10.1016/j.jaci.2018.05.014
DO - 10.1016/j.jaci.2018.05.014
M3 - Article
C2 - 29852258
AN - SCOPUS:85049074301
SN - 0091-6749
VL - 142
SP - 130-138.e1
JO - Journal of Allergy and Clinical Immunology
JF - Journal of Allergy and Clinical Immunology
IS - 1
ER -