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

In corso di stampa. Prove corrette dall'autore. Disponibile online dal Thursday 22 August 2024
This article has been published in an issue click here to access

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.

Il testo completo di questo articolo è disponibile in PDF.

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

Mappa

Esportazione

Benvenuto su EM|consulte, il riferimento dei professionisti della salute.
L'accesso al testo integrale di questo articolo richiede un abbonamento.

Già abbonato a @@106933@@ rivista ?

connettersi o creare un account

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

Abstract

Background

Objective

Methods

Results

Conclusions

Mappa

Citazioni Export

File

Contenuto

Il mio account

Aide & support

Dichiarazione CNIL