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度普利尤单抗对中重度特应性皮炎幼儿的照料者及患者报告结局的影响:一项安慰剂对照的3期研究结果

The effect of dupilumab on caregiver- and patient-reported outcomes in young children with moderate-to-severe atopic dermatitis: Results from a placebo-controlled, phase 3 study.

作者信息

Paller Amy S, Silverberg Jonathan I, Simpson Eric L, Cork Michael J, Arkwright Peter D, Chen Zhen, Bansal Ashish, Prescilla Randy, Wang Zhixiao, Marco Ainara R

机构信息

Department of Dermatology, Northwestern University Feinberg School of Medicine, Chicago, Illinois; Division of Dermatology, Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois.

Department of Dermatology, The George Washington University School of Medicine and Health Sciences, Washington, District of Columbia.

出版信息

J Am Acad Dermatol. 2025 Jan;92(1):116-126. doi: 10.1016/j.jaad.2024.09.039. Epub 2024 Sep 28.

Abstract

BACKGROUND

Moderate-to-severe atopic dermatitis (AD) greatly impacts children/caregivers.

OBJECTIVE

Evaluate the impact of treatment with dupilumab on caregiver- and patient-reported AD symptoms and quality of life (QoL) in young children.

METHODS

In the LIBERTY AD PRESCHOOL (randomized, placebo-controlled) study, children aged 6 months to 5 years with moderate-to-severe AD received dupilumab or placebo plus low-potency topical corticosteroids for 16 weeks. This posthoc analysis assessed the change from baseline to week 16 in caregiver-reported outcome measures of AD symptoms (eg, itch and sleep) and QoL of patients and their caregivers/families.

RESULTS

Dupilumab (n = 83) vs placebo (n = 79) provided significant improvements in caregiver-reported AD symptoms and QoL. Significant improvements were seen as early as week 4 and sustained through the end of the study. Additionally, dupilumab vs placebo provided rapid and significant improvement in QoL measures for the patients' caregivers/families.

LIMITATIONS

Few patients aged <2 years; significance only reported for prespecified endpoints; Infant's Dermatitis QoL Index severity strata adopted from Children's Dermatology Life Quality Index.

CONCLUSION

Dupilumab improved AD symptoms and QoL in patients and their caregivers/families.

摘要

背景

中重度特应性皮炎(AD)对儿童/照料者有极大影响。

目的

评估度普利尤单抗治疗对幼儿照料者及患者报告的AD症状和生活质量(QoL)的影响。

方法

在LIBERTY AD PRESCHOOL(随机、安慰剂对照)研究中,6个月至5岁的中重度AD患儿接受度普利尤单抗或安慰剂加低效局部糖皮质激素治疗16周。这项事后分析评估了从基线到第16周照料者报告的AD症状(如瘙痒和睡眠)以及患者及其照料者/家庭的生活质量的结局指标的变化。

结果

度普利尤单抗组(n = 83)与安慰剂组(n = 79)相比,照料者报告的AD症状和生活质量有显著改善。早在第4周就出现了显著改善,并持续到研究结束。此外,度普利尤单抗组与安慰剂组相比,患者照料者/家庭的生活质量指标有快速且显著的改善。

局限性

2岁以下患者很少;仅报告了预设终点的显著性;婴儿性皮炎生活质量指数严重程度分层采用儿童皮肤病生活质量指数。

结论

度普利尤单抗改善了患者及其照料者/家庭的AD症状和生活质量。

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