Long-term durability between parent and child patient-reported outcomes in eosinophilic esophagitis - 22/08/24

Doi : 10.1016/j.jaci.2024.07.011

Lisa J. Martin, PhD ^a,^b, Xue Zhang, PhD ^c, Mirna Chehade, MD ^d, Carla M. Davis, MD ^e,^f, Evan S. Dellon, MD, MPH ^g,^h, Gary W. Falk, MD ⁱ, Sandeep K. Gupta, MD ^j, Ikuo Hirano, MD ^k, Girish S. Hiremath, MD ^l, David A. Katzka, MD ^m, Paneez Khoury, MD ⁿ, John Leung, MD ^o,^p, Paul Menard-Katcher, MD ^q, Nirmala Gonsalves, MD ^k, Robert D. Pesek, MD ^r,^s, Jonathan M. Spergel, MD, PhD ^t,^u, Joshua B. Wechsler, MD ^v, Kara Kliewer, PhD ^c, Nicoleta C. Arva, MD ^w, Margaret H. Collins, MD ^a,^b, Maria Pletneva, MD ^x, Guang-Yu Yang, MD ^y, Glenn T. Furuta, MD ^z,^aa, Marc E. Rothenberg, MD, PhD ^bb, Seema S. Aceves, MD, PhD ^cc,^⁎
^a Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
^b University of Cincinnati College of Medicine, Cincinnati, Ohio
^c Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
^d Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY
^e Division of Immunology, Allergy, and Retrovirology, Baylor College of Medicine, Texas Children’s Hospital, Houston, Tex
^f Food Allergy Program, Texas Children’s Hospital, Houston, Tex
^g Center for Esophageal Diseases and Swallowing, Center for Gastrointestinal Biology and Disease, University of North Carolina School of Medicine, Chapel Hill, NC
^h Division of Gastroenterology and Hepatology, Center for Gastrointestinal Biology and Disease, University of North Carolina School of Medicine, Chapel Hill, NC
ⁱ Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pa
^j Children’s of Alabama, University of Alabama at Birmingham, Birmingham, Ala
^k Division of Gastroenterology and Hepatology, Kenneth Griffin Esophageal Center, Northwestern University Feinberg School of Medicine, Chicago, Ill
^l Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Vanderbilt University Medical Center, Nashville, Tenn
^m Division of Digestive and Liver Diseases, Columbia University Irving Medical Center, New York, NY
ⁿ National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md
^o Division of Allergy/Immunology, Tuft’s Medical Center, Boston, Mass
^p Division of Gastroenterology, Tuft’s Medical Center, Boston, Mass
^q University of Colorado School of Medicine, Anschutz Medical Campus, Aurora, Colo
^r Division of Allergy/Immunology, University of Arkansas for Medicine Sciences, Little Rock, Ark
^s Arkansas Children’s Hospital, Little Rock, Ark
^t Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pa
^u Division of Allergy and Immunology, Children’s Hospital of Philadelphia, Philadelphia, Pa
^v Division of Gastroenterology, Hepatology and Nutrition, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, Ill
^w Department of Pathology, Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Ill
^x Department of Pathology, University of Utah, Salt Lake City, Utah
^y Department of Pathology, Northwestern University Feinberg School of Medicine, Chicago, Ill
^z Section of Pediatric Gastroenterology, Hepatology and Nutrition, Digestive Health Institute, Children’s Hospital of Colorado, Aurora, Colo
^aa Gastrointestinal Eosinophilic Disease Program, Mucosal Inflammation Program, University of Colorado School of Medicine, Aurora, Colo
^bb Division of Allergy and Immunology, Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine, Cincinnati, Ohio
^cc University of California San Diego, Rady Children’s Hospital of San Diego, San Diego, Calif

^∗Corresponding author: Seema S. Aceves, MD, PhD, Divison of Allergy, Immunology, University of California, San Diego, 3147 Biomedical Sciences Way, La Jolla, CA 92093.3147 Biomedical Sciences WayLa JollaCA92093

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Abstract

Background

Because young children cannot self-report symptoms, there is a need for parent surrogate reports. Although early work suggested parent-child alignment for eosinophil esophagitis (EoE) patient-reported outcomes (PROs), the longitudinal alignment is unclear.

Objective

We sought to assess the agreement and longitudinal stability of PROs between children with EoE and their parents.

Methods

A total of 292 parent-child respondents completed 723 questionnaires over 5 years in an observational trial in the Consortium of Eosinophilic Gastrointestinal Disease Researchers. The change in and agreement between parent and child Pediatric Eosinophilic Esophagitis Symptom Score version 2 (PEESSv2.0) and Pediatric Quality of Life Eosinophilic Esophagitis Module (PedsQL-EoE) PROs over time were assessed using Pearson correlation and Bland-Altman analyses. Clinical factors influencing PROs and their agreement were evaluated using linear mixed models.

Results

The cohort had a median disease duration equaling 3.7 years and was predominantly male (73.6%) and White (85.3%). Child and parent PEESSv2.0 response groups were identified and were stable over time. There was strong correlation between child and parent reports (PEESSv2.0, 0.83;PedsQL-EoE, 0.74), with minimal pairwise differences for symptoms. Longitudinally, parent-reported PedsQL-EoE scores were stable (P ≥ .32), whereas child-reported PedsQL-EoE scores improved (P = .026). A larger difference in parent and child PedsQL-EoE reports was associated with younger age (P < .001), and differences were driven by psychosocial PRO domains.

Conclusions

There is strong longitudinal alignment between child and parent reports using EoE PROs. These data provide evidence that parent report is a stable proxy for objective EoE symptoms in their children.

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Key words : Pediatric, clinical trial readiness, eosinophilic esophagitis, food allergy, multicenter, patient-reported outcomes, quality of life

Abbreviations used : CEGIR, EoE, FDA, IQR, NIAID, PedsQL-EoE, PEESSv2.0, PRO, QoL