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儿童和父母报告的嗜酸性食管炎患者结局的长期耐久性。

Long-term durability between parent and child patient-reported outcomes in eosinophilic esophagitis.

机构信息

Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; University of Cincinnati College of Medicine, Cincinnati, Ohio.

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.

出版信息

J Allergy Clin Immunol. 2024 Nov;154(5):1232-1240.e12. doi: 10.1016/j.jaci.2024.07.011. Epub 2024 Jul 25.


DOI:10.1016/j.jaci.2024.07.011
PMID:39059504
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11879065/
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.

摘要

背景:由于幼儿无法自我报告症状,因此需要家长作为替代者进行报告。尽管早期的研究表明,父母-子女对嗜酸性食管炎(EoE)患者报告的结果(PRO)的一致性,但纵向一致性尚不清楚。

目的:我们旨在评估 EoE 患儿及其父母的 PRO 之间的一致性和纵向稳定性。

方法:在过敏性胃肠道疾病研究联合会的一项观察性试验中,共有 292 对父母-子女受访者在 5 年内完成了 723 份调查问卷。使用 Pearson 相关分析和 Bland-Altman 分析评估了儿童和父母的儿科嗜酸性食管炎症状评分 2.0 版(PEESSv2.0)和儿科生活质量 EoE 模块(PedsQL-EoE)PRO 随时间的变化和一致性。使用线性混合模型评估了影响 PRO 及其一致性的临床因素。

结果:该队列的疾病持续时间中位数为 3.7 年,主要为男性(73.6%)和白人(85.3%)。确定了儿童和父母的 PEESSv2.0 反应组,并且这些组随时间保持稳定。儿童和父母报告之间存在很强的相关性(PEESSv2.0,0.83;PedsQL-EoE,0.74),症状之间的差异最小。纵向来看,父母报告的 PedsQL-EoE 评分稳定(P≥0.32),而儿童报告的 PedsQL-EoE 评分有所提高(P=0.026)。父母和儿童的 PedsQL-EoE 报告差异较大与年龄较小有关(P<0.001),差异主要由心理社会 PRO 领域驱动。

结论:使用 EoE PRO,儿童和父母报告之间存在很强的纵向一致性。这些数据为父母报告是其子女客观 EoE 症状的稳定替代指标提供了证据。

相似文献

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Long-term durability between parent and child patient-reported outcomes in eosinophilic esophagitis.

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[3]
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[4]
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[5]
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[6]
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[7]
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[9]
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[10]
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Turk J Gastroenterol. 2021-4

本文引用的文献

[1]
Esophageal Remodeling Correlates With Eating Behaviors in Pediatric Eosinophilic Esophagitis.

Am J Gastroenterol. 2024-6-1

[2]
The Index of Severity for Eosinophilic Esophagitis (I-SEE) Reflects Longitudinal Clinicopathologic Changes in Children.

Clin Gastroenterol Hepatol. 2024-4

[3]
Efficacy and safety of dupilumab up to 52 weeks in adults and adolescents with eosinophilic oesophagitis (LIBERTY EoE TREET study): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial.

Lancet Gastroenterol Hepatol. 2023-11

[4]
A Newly Proposed Severity Index for Eosinophilic Esophagitis is Associated With Baseline Clinical Features and Successful Treatment Response.

Clin Gastroenterol Hepatol. 2023-9

[5]
Dupilumab in Patients with Eosinophilic Esophagitis. Reply.

N Engl J Med. 2023-3-9

[6]
A Clinical Severity Index for Eosinophilic Esophagitis: Development, Consensus, and Future Directions.

Gastroenterology. 2022-7

[7]
Close follow-up is associated with fewer stricture formation and results in earlier detection of histological relapse in the long-term management of eosinophilic esophagitis.

United European Gastroenterol J. 2022-4

[8]
Profile Comparison of Patient-Reported and Proxy-Reported Symptoms in Pediatric Patients With Cancer Receiving Chemotherapy.

JAMA Netw Open. 2022-3-1

[9]
Optimal Methods for Reducing Proxy-Introduced Bias on Patient-Reported Outcome Measurements for Group-Level Analyses.

Circ Cardiovasc Qual Outcomes. 2021-11

[10]
The use of proxies and proxy-reported measures: a report of the international society for quality of life research (ISOQOL) proxy task force.

Qual Life Res. 2022-2

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