12岁以下儿童中重度特应性皮炎全身治疗的真实世界治疗结果：特应性皮炎儿科研究的2年结果

Real-world treatment outcomes of systemic treatments for moderate-to-severe atopic dermatitis in children aged less than 12 years: 2-year results from PEDIatric STudy in Atopic Dermatitis.

作者信息

Paller Amy S, de Bruin-Weller Marjolein, Marcoux Danielle, Baselga Eulalia, Oliveira de Carvalho Vania, Ardusso Ledit R F, Pasmans Suzanne G M A, Toledo-Bahena Mirna, Rubin Cory, Joyce Joel C, Wine Lee Lara, Adams Bryan, Gupta Rajan, Ardeleanu Marius, Zhang Annie

机构信息

Departments of Dermatology and Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

National Expertise Center for Atopic Dermatitis, Department of Dermatology and Allergology, University Medical Center Utrecht, Utrecht, The Netherlands.

出版信息

J Am Acad Dermatol. 2025 Feb;92(2):242-251. doi: 10.1016/j.jaad.2024.09.046. Epub 2024 Oct 9.

DOI:10.1016/j.jaad.2024.09.046

PMID:39389429

Abstract

BACKGROUND

The arrival of biologics and small-molecule therapies (eg Janus kinase inhibitors) changed atopic dermatitis treatment, but older systemic treatments continue to be prescribed.

OBJECTIVE

To provide real-world effectiveness, safety, and adherence data for dupilumab, cyclosporine, and methotrexate.

METHODS

PEDIatric STudy in Atopic Dermatitis (NCT03687359) is a real-world, prospective, observational, 10-year study of children (<12 years) with inadequately controlled moderate-to-severe atopic dermatitis. We report 2-year interim results.

RESULTS

Median treatment durations were 8.1, 13.0, and 10.7 months for dupilumab (n = 144), methotrexate (n = 114), and cyclosporine (n = 121), respectively. Dupilumab had numerically greater within-group improvements than methotrexate and cyclosporine in Eczema Area and Severity Index (-12.4∗ vs -5.7∗ and -3.3); body surface area affected (-19.9%∗ vs -11.8%∗ and -8.8%∗); itching (night-time: -2.1∗ vs -0.4 and + 0.1; daytime: -1.5∗ vs +0.1 and + 0.2; ≥6 years); itching/scratching (-3.6∗ vs -1.4∗ and -0.2; <6 years); and Patient-Oriented Eczema Measure (-7.0∗ vs -4.7∗ and -1.5) (∗P < .05 within-group improvements from baseline). Dupilumab had less discontinuations (8.3% vs 28.9% and 43.0%) and adverse event(s) (18.1% vs 29.8% and 31.4%).

LIMITATIONS

No randomization, placebo, or specified dosages.

CONCLUSION

Dupilumab was associated with numerically greater outcomes and higher adherence than cyclosporine or methotrexate.

摘要

背景

生物制剂和小分子疗法（如 Janus 激酶抑制剂）的出现改变了特应性皮炎的治疗方式，但 older 全身治疗仍在继续被处方使用。

目的

提供度普利尤单抗、环孢素和甲氨蝶呤的真实世界有效性、安全性和依从性数据。

方法

儿童特应性皮炎研究（NCT03687359）是一项针对中度至重度特应性皮炎控制不佳的 12 岁以下儿童进行的为期 10 年的真实世界、前瞻性、观察性研究。我们报告 2 年中期结果。

结果

度普利尤单抗（n = 144）、甲氨蝶呤（n = 114）和环孢素（n = 121）的中位治疗持续时间分别为 8.1、13.0 和 10.7 个月。度普利尤单抗在湿疹面积和严重程度指数（-12.4∗ 对 -5.7∗ 和 -3.3）、受影响的体表面积（-19.9%∗ 对 -11.8%∗ 和 -8.8%∗）、瘙痒（夜间：-2.1∗ 对 -0.4 和 +0.1；白天：-1.5∗ 对 +0.1 和 +0.2；≥6 岁）、瘙痒/抓挠（-3.6∗ 对 -1.4∗ 和 -0.2；<6 岁）以及患者导向性湿疹测量（-7.0∗ 对 -4.7∗ 和 -1.5）方面的组内改善在数值上大于甲氨蝶呤和环孢素（∗P <.05，组内相对于基线的改善）。度普利尤单抗的停药率（8.3% 对 28.9% 和 43.0%）和不良事件发生率（18.1% 对 29.8% 和 31.4%）更低。