TY - JOUR
T1 - Patient-Reported Outcome Scores Are Comparable in Multiregional Versus Uniregional Eosinophilic Esophagitis
AU - Collins, Margaret H.
AU - Pan, Zhaoxing
AU - Yang, Guang Yu
AU - Arva, Nicoleta C.
AU - Pletneva, Maria A.
AU - Martin, Lisa J.
AU - Aceves, Seema
AU - Chehade, Mirna
AU - Dellon, Evan S.
AU - Spergel, Jonathan M.
AU - Menard-Katcher, Paul
AU - Peterson, Kathryn A.
AU - Khoury, Paneez
AU - Gonsalves, Nirmala
AU - Gupta, Sandeep K.
AU - Davis, Carla M.
AU - Falk, Gary W.
AU - Wechsler, Joshua B.
AU - Pesek, Robert
AU - Hiremath, Girish
AU - Leung, John
AU - Katzka, David A.
AU - Furuta, Glenn T.
AU - Rothenberg, Marc E.
N1 - Publisher Copyright:
© 2025 The Authors
PY - 2025/1
Y1 - 2025/1
N2 - Background and Aims: Eosinophilic esophagitis (EoE) clinical trials often require multiregional esophageal inflammation. To determine if patient-reported outcomes (PROs) worsen with multiregional eosinophil inflammation (≥15 eosinophils/high power field), we compared outcomes scores when multiple (proximal/mid/distal) esophageal regions, versus one, were inflamed. Methods: The Consortium of Eosinophilic Gastrointestinal Disorders Researchers database was searched for peak eosinophil counts (PECs), eosinophilic esophagitis histology scoring system scores, and PRO scores (eosinophilic esophagitis activity index; Pediatric Eosinophilic Esophagitis Symptom Score; EoE quality of life; Pediatric Quality of Life Eosinophilic Esophagitis modulev3.0) in submissions from one or more regions. Analyses were performed with unadjusted or adjusted (adults: age, sex, dilation within 1 year; children: age, sex) data, using Wilcoxon rank sum and T test, and least squares mean, respectively. An interaction test was used for subgroup analysis. P ≤ .05 was considered significant. Results: Adult PEC was 60.2 ± 44.8 vs 39.5 ± 29.9 (mean ± standard deviation, P < .004), child PEC was 66.4 ± 38.2 vs 38.2 ± 31.5 (P = .0007) when two or more regions, vs one, were inflamed, respectively. Most symptoms and QoL scores did not differ when two or more regions (67 adults, 17 children) vs one (48 adults, 27 children) were inflamed: exceptions were modest worsening of one adult symptom domain (adjusted avoidance/modification/slow eating), and one child Pediatric QL Eosinophilic Esophagitis modulev3.0 domain (chest pain/heartburn/stomach aches/nausea/vomiting/food regurgitation) (each P < .043). Eosinophilic esophagitis histology scoring system scores were significantly increased with multiregional inflammation in adults and children (all P < .01), and scores correlated significantly with symptoms in one uniregional group (0.29–0.38, P < .01 to 0.05) but not any multiregional group. Conclusion: Multiregional compared to uniregional esophageal eosinophil inflammation does not significantly impact most PRO scores, and may not be necessary for all clinical trials.
AB - Background and Aims: Eosinophilic esophagitis (EoE) clinical trials often require multiregional esophageal inflammation. To determine if patient-reported outcomes (PROs) worsen with multiregional eosinophil inflammation (≥15 eosinophils/high power field), we compared outcomes scores when multiple (proximal/mid/distal) esophageal regions, versus one, were inflamed. Methods: The Consortium of Eosinophilic Gastrointestinal Disorders Researchers database was searched for peak eosinophil counts (PECs), eosinophilic esophagitis histology scoring system scores, and PRO scores (eosinophilic esophagitis activity index; Pediatric Eosinophilic Esophagitis Symptom Score; EoE quality of life; Pediatric Quality of Life Eosinophilic Esophagitis modulev3.0) in submissions from one or more regions. Analyses were performed with unadjusted or adjusted (adults: age, sex, dilation within 1 year; children: age, sex) data, using Wilcoxon rank sum and T test, and least squares mean, respectively. An interaction test was used for subgroup analysis. P ≤ .05 was considered significant. Results: Adult PEC was 60.2 ± 44.8 vs 39.5 ± 29.9 (mean ± standard deviation, P < .004), child PEC was 66.4 ± 38.2 vs 38.2 ± 31.5 (P = .0007) when two or more regions, vs one, were inflamed, respectively. Most symptoms and QoL scores did not differ when two or more regions (67 adults, 17 children) vs one (48 adults, 27 children) were inflamed: exceptions were modest worsening of one adult symptom domain (adjusted avoidance/modification/slow eating), and one child Pediatric QL Eosinophilic Esophagitis modulev3.0 domain (chest pain/heartburn/stomach aches/nausea/vomiting/food regurgitation) (each P < .043). Eosinophilic esophagitis histology scoring system scores were significantly increased with multiregional inflammation in adults and children (all P < .01), and scores correlated significantly with symptoms in one uniregional group (0.29–0.38, P < .01 to 0.05) but not any multiregional group. Conclusion: Multiregional compared to uniregional esophageal eosinophil inflammation does not significantly impact most PRO scores, and may not be necessary for all clinical trials.
KW - Clinical care
KW - Clinical disease metrics
KW - Diagnosis
KW - Symptom correlation
KW - Ttesting clinical trial enrollment assumptions
UR - https://www.scopus.com/pages/publications/105015088312
U2 - 10.1016/j.gastha.2025.100753
DO - 10.1016/j.gastha.2025.100753
M3 - Article
AN - SCOPUS:105015088312
SN - 2772-5723
VL - 4
JO - Gastro Hep Advances
JF - Gastro Hep Advances
IS - 9
M1 - 100753
ER -