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巴瑞替尼联合外用糖皮质激素治疗对外用糖皮质激素反应不佳的中重度特应性皮炎儿科患者的疗效和安全性:一项III期、随机、双盲、安慰剂对照研究(BREEZE-AD PEDS)的结果

Efficacy and safety of baricitinib in combination with topical corticosteroids in paediatric patients with moderate-to-severe atopic dermatitis with an inadequate response to topical corticosteroids: results from a phase III, randomized, double-blind, placebo-controlled study (BREEZE-AD PEDS).

作者信息

Torrelo Antonio, Rewerska Barbara, Galimberti Maria, Paller Amy, Yang Chin-Yi, Prakash Apurva, Zhu Danting, Pontes Filho Marco Antonio G, Wu Wen-Shuo, Eichenfield Lawrence F

机构信息

Department of Dermatology, Hospital Infantil Universitario Niño Jesús, Madrid, Spain.

Diamond Clinic, Kraków, Poland.

出版信息

Br J Dermatol. 2023 Jul 7;189(1):23-32. doi: 10.1093/bjd/ljad096.

Abstract

BACKGROUND

Baricitinib, an oral selective Janus kinase (JAK)1/JAK2 inhibitor, is approved in many countries for moderate-to-severe atopic dermatitis (AD) in adults who are candidates for systemic therapy.

OBJECTIVES

To evaluate the efficacy and safety of three doses of baricitinib in combination with low-to-moderate potency topical corticosteroids in paediatric patients with moderate-to-severe AD.

METHODS

Patients (aged 2 to < 18 years) were randomized (1 : 1 : 1 : 1) to once-daily baricitinib low dose (1 mg equivalent), medium dose (2 mg equivalent), high dose (4 mg equivalent) or placebo for 16 weeks. The primary endpoint was the proportion of patients achieving a validated Investigator Global Assessment® (vIGA-AD) of 0/1 with a ≥ 2-point improvement at week 16. Key secondary endpoints included the proportions of patients achieving ≥ 75% and ≥ 90% improvement in the Eczema Area and Severity Index (EASI-75 and EASI-90, respectively), ≥ 75% improvement in the SCORing Atopic Dermatitis (SCORAD 75), mean change from baseline in EASI score and proportion of patients achieving a 4-point improvement in the Itch Numeric Rating scale (NRS) for patients aged ≥ 10 years. Primary and key secondary efficacy analyses were conducted on the intent-to-treat population and adjusted for multiplicity. Safety analyses included all randomized patients who received ≥ 1 dose of study treatment.

RESULTS

A total of 483 patients were randomized (mean age 12 years). The baricitinib 4 mg equivalent achieved a statistically significant (P < 0.05) improvement vs. placebo on all 16-week endpoints (vIGA 0/1 with ≥ 2-point improvement, EASI-75, EASI-90, SCORAD 75, mean change in EASI score and Itch NRS 4-point improvement for patients aged ≥ 10 years). Improvement (P < 0.05, non-multiplicity adjusted) was also observed for baricitinib 4 mg equivalent vs. placebo in the ability to fall asleep and in reduction of topical corticosteroid use. Few patients discontinued due to adverse events (1.6% for placebo and 0.6% for those treated with baricitinib). There were no deaths, venous thromboembolic events, arterial thrombotic events, major adverse cardiovascular events, malignancies, gastrointestinal perforations or opportunistic infections seen.

CONCLUSIONS

The results indicate that baricitinib offers a potential therapeutic option with a favourable benefit-risk profile for paediatric patients with moderate-to-severe AD who are candidates for systemic therapies.

摘要

背景

巴瑞替尼是一种口服选择性 Janus 激酶(JAK)1/JAK2 抑制剂,在许多国家被批准用于适合进行全身治疗的成人中重度特应性皮炎(AD)。

目的

评估三种剂量的巴瑞替尼联合低至中效外用糖皮质激素治疗中重度 AD 儿科患者的疗效和安全性。

方法

将患者(年龄 2 至<18 岁)按 1:1:1:1 随机分为每日一次的巴瑞替尼低剂量(1mg 等效剂量)、中剂量(2mg 等效剂量)、高剂量(4mg 等效剂量)或安慰剂组,治疗 16 周。主要终点是在第 16 周达到有效研究者整体评估(vIGA-AD)为 0/1 且改善≥2 分的患者比例。关键次要终点包括达到湿疹面积和严重程度指数改善≥75%和≥90%(分别为 EASI-75 和 EASI-90)、特应性皮炎评分(SCORAD)改善≥75%、EASI 评分相对于基线的平均变化以及年龄≥10 岁患者的瘙痒数字评定量表(NRS)改善 4 分的患者比例。主要和关键次要疗效分析在意向性治疗人群中进行,并针对多重性进行调整。安全性分析包括所有接受≥1 剂研究治疗的随机分组患者。

结果

共 483 例患者被随机分组(平均年龄 12 岁)。在所有 16 周的终点指标上(vIGA 0/1 且改善≥2 分、EASI-75、EASI-90、SCORAD 75、EASI 评分的平均变化以及年龄≥10 岁患者的瘙痒 NRS 改善 4 分),4mg 等效剂量的巴瑞替尼与安慰剂相比取得了具有统计学意义(P<0.05)的改善。在入睡能力和减少外用糖皮质激素使用方面,4mg 等效剂量的巴瑞替尼与安慰剂相比也观察到改善(P<0.05,未进行多重性调整)。很少有患者因不良事件停药(安慰剂组为 1.6%,巴瑞替尼治疗组为 0.6%)。未观察到死亡、静脉血栓栓塞事件、动脉血栓形成事件、主要不良心血管事件、恶性肿瘤、胃肠道穿孔或机会性感染。

结论

结果表明,对于适合进行全身治疗的中重度 AD 儿科患者,巴瑞替尼提供了一种潜在的治疗选择,其效益风险比良好。

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