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度普利尤单抗在不同表型和内型特应性皮炎儿童中的疗效:病例系列

Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series.

作者信息

Rossi Ana B, Mello Adriana M, Zahn Joseph

机构信息

Sanofi, 450 Water Street, Cambridge, MA, 02141, USA.

Regeneron Pharmaceuticals Inc., Tarrytown, NY, USA.

出版信息

Adv Ther. 2025 May 8. doi: 10.1007/s12325-025-03150-6.

Abstract

INTRODUCTION

Atopic dermatitis (AD) is a prevalent disease in infants and young children worldwide. Dupilumab has been shown to rapidly and significantly improve AD signs, symptoms, and quality of life in pediatric patients with moderate-to-severe AD.

METHODS

In LIBERTY AD PRESCHOOL, a randomized, double-blind, placebo-controlled, phase 3 clinical trial, patients aged 6 months to 5 years with moderate-to-severe AD received subcutaneous dupilumab or matched placebo every 4 weeks for 16 weeks. All patients received concomitant low-potency topical corticosteroids. Here, we present 12 photographic cases of patients with different phenotypes and endotypes in the dupilumab group, representative of the study population, before and after treatment. Each case is presented with clinical outcome measures of AD severity and quality of life, as well as relevant biomarkers, with percent improvement from baseline.

RESULTS

Treatment with dupilumab led to visible improvements in signs of lichenification, erythema, excoriations, skin dryness, and oozing/crusting. Clinically meaningful improvements in the measured outcomes were observed in most of the patients, including AD extent and severity, clinical lesions, itch, sleep loss, frequency of symptoms, and quality of life. These improvements were associated with substantial reductions in AD-related biomarkers.

CONCLUSION

Treatment with dupilumab improved signs, symptoms, and quality of life, and reduced AD-related serum biomarkers in young children with moderate-to-severe AD with different phenotypes and endotypes.

TRIAL REGISTRATION

The trial was registered with ClinicalTrials.gov with ID number NCT03346434 on November 17, 2017. Video abstract available for this article. Video abstract (MP4 1,02,609 KB).

摘要

引言

特应性皮炎(AD)是一种在全球婴幼儿中普遍存在的疾病。已证明度普利尤单抗能迅速且显著改善中度至重度AD儿科患者的AD体征、症状和生活质量。

方法

在LIBERTY AD PRESCHOOL这一随机、双盲、安慰剂对照的3期临床试验中,年龄在6个月至5岁的中度至重度AD患者每4周接受一次皮下注射度普利尤单抗或匹配的安慰剂,共治疗16周。所有患者均同时接受低效局部皮质类固醇治疗。在此,我们展示了度普利尤单抗组中12例具有不同表型和内型的患者在治疗前后的照片病例,这些病例代表了研究人群。每个病例都呈现了AD严重程度和生活质量的临床结局指标,以及相关生物标志物,并列出了相对于基线的改善百分比。

结果

度普利尤单抗治疗使苔藓化、红斑、抓痕、皮肤干燥和渗出/结痂等体征有明显改善。在大多数患者中观察到所测量结局有临床意义的改善,包括AD范围和严重程度、临床皮损、瘙痒、睡眠障碍、症状发作频率和生活质量。这些改善与AD相关生物标志物的大幅减少有关。

结论

度普利尤单抗治疗改善了中度至重度AD且具有不同表型和内型的幼儿的体征、症状和生活质量,并降低了AD相关血清生物标志物。

试验注册

该试验于2017年11月17日在ClinicalTrials.gov注册,注册号为NCT03346434。本文有视频摘要。视频摘要(MP4格式,1,02,609KB)。

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