Alignment of parent- and child-reported outcomes and histology in eosinophilic esophagitis across multiple CEGIR sites

被引：48

作者：

Aceves, Seema S. ^{[1
]}

King, Eileen ^{[2
]}

Collins, Margaret H. ^{[3
]}

Yang, Guang-Yu ^{[4
]}

Capocelli, Kelley E. ^{[6
]}

Abonia, J. Pablo ^{[8
]}

Atkins, Dan ^{[7
]}

Bonis, Peter A. ^{[9
]}

Carpenter, Christina L. ^{[10
,11
]}

Dellon, Evan S. ^{[12
]}

Eby, Michael D. ^{[8
]}

Falk, Gary W. ^{[13
]}

Gonsalves, Nirmala ^{[5
]}

Gupta, Sandeep K. ^{[14
]}

Hirano, Ikuo ^{[5
]}

Kocher, Kendra ^{[15
]}

Krischer, Jeffrey P. ^{[10
,11
]}

Leung, John ^{[9
]}

Lipscomb, Jessi ^{[2
]}

Menard-Katcher, Paul ^{[15
]}

Mukkada, Vincent A. ^{[16
]}

Pan, Zhaoxing ^{[17
]}

Spergel, Jonathan M. ^{[18
]}

Sun, Qin ^{[2
]}

Wershil, Barry K. ^{[19
]}

Rothenberg, Marc E. ^{[8
]}

Furuta, Glenn T. ^{[15
]}

机构：

[1] Univ Calif San Diego, Div Allergy Immunol, Rady Childrens Hosp, San Diego, CA 92103 USA

[2] Univ Cincinnati, Coll Med, Dept Pediat,Div Biostat & Epidemiol, Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[3] Univ Cincinnati, Coll Med, Dept Pediat, Div Pathol, Cincinnati, OH USA

[4] Northwestern Univ, Feinberg Sch Med, Div Pathol, Chicago, IL 60611 USA

[5] Northwestern Univ, Feinberg Sch Med, Div Gastroenterol & Hepatol, Chicago, IL 60611 USA

[6] Childrens Hosp Colorado, Div Pathol, Aurora, CO USA

[7] Childrens Hosp Colorado, Sect Allergy, Immunol, Aurora, CO USA

[8] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp Med Ctr, Div Allergy & Immunol, 3333 Burnet Ave, Cincinnati, OH 45229 USA

[9] Tufts Med Ctr, Div Gastroenterol, Boston, MA USA

[10] Univ S Florida, Morsani Coll Med, Hlth Informat Inst, Dept Pediat, Tampa, FL USA

[11] Univ S Florida, Morsani Coll Med, Hlth Informat Inst, Dept Med, Tampa, FL USA

[12] Univ N Carolina, Dept Gastroenterol & Hepatol, Sch Med, Chapel Hill, NC 27515 USA

[13] Univ Penn, Perelman Sch Med, Div Gastroenterol, Philadelphia, PA 19104 USA

[14] Univ Illinois, Coll Med, Dept Pediat Gastroenterol Hepatol & Nutr, Chicago, IL USA

[15] Univ Colorado, Childrens Hosp Colorado, Gastrointestinal Eosinophil Dis Program, Sect Pediat Gastroenterol Hepatol & Nutr,Sch Med, Aurora, CO USA

[16] Univ Cincinnati, Coll Med, Dept Pediat,Div Gastroenterol Hepatol & Nutr, Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[17] Childrens Hosp Colorado, Pediat Gastroenterol Pediat Allergy & Immunol, Aurora, CO USA

[18] Univ Penn, Childrens Hosp Philadelphia, Dept Allergy & Immunol, Perelman Sch Med, Philadelphia, PA 19104 USA

[19] Ann & Robert H Lurie Childrens Hosp Chicago, Div Gastroenterol Hepatol & Nutr, Chicago, IL 60611 USA

来源：

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY | 2018年 / 142卷 / 01期

关键词：

Eosinophil; eosinophilic esophagitis; eosinophilic oesophagitis; Consortium of Eosinophilic Gastrointestinal Disease Researchers; patient-reported outcomes; pediatric eosinophilic esophagitis symptom score; version; 2; pediatric QOL EoE module; symptoms; quality of life; SYMPTOMS; DISEASE; LONG;

D O I：

10.1016/j.jaci.2018.05.014

中图分类号：

R392 [医学免疫学];

学科分类号：

100102 ;

摘要：

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 5 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.

引用

页码：130 / +

页数：10

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