奥马珠单抗治疗儿童过敏性哮喘：来自随机试验荟萃分析的有效性与安全性循证见解。
完整摘要 · 中文译文
目的：本荟萃分析旨在综合英文随机对照试验（RCT）的数据，以评估奥马珠单抗在6～20岁过敏性哮喘患者中的疗效和安全性，并在可行的情况下特别关注按年龄分层的结果。 方法：本综述按照PRISMA 2020指南进行，并遵循预先注册的研究方案（PROSPERO：CRD420251132811）。符合条件的研究为英文RCT，主要纳入儿童和青少年过敏性哮喘患者；其中一项纳入的试验招募了年龄最大至20岁的受试者，但仅有15名受试者（占总样本的0.8%）年龄在18～20岁之间，其余所有受试者均<18岁。研究将奥马珠单抗与对照治疗（安慰剂或标准治疗）进行比较。疗效结局包括哮喘急性发作、第1秒用力呼气容积（FEV1）和儿童哮喘控制测试（C-ACT）评分。安全性结局包括治疗期间出现的不良事件（TEAE）、严重不良事件（SAE）和哮喘相关住院。在适当情况下使用随机效应模型合并数据，并通过I²统计量评估异质性。 结果：共纳入6项RCT，涉及1,835名年龄在6～20岁的受试者（奥马珠单抗组：1,134名；对照组：701名）。奥马珠单抗显著减少了哮喘急性发作（相对危险度 RR = 0.66，95% CI：0.55～0.79；I² = 51.6%）、SAE（RR = 0.56，95% CI：0.35～0.90；I² = 43.1%）和住院（RR = 0.35，95% CI：0.22～0.56；I² = 0.0%）。在TEAE方面未发现显著差异（RR = 0.96，95 % CI：0.92-1.01；I2 = 1.5%）。关于肺功能和哮喘控制的证据有限，无法进行定量合并。 结论：奥马珠单抗可有效减少儿童和青少年过敏性哮喘的急性发作、严重不良事件和住院，且安全性良好。关于肺功能和哮喘控制的证据仍然有限，应谨慎解读。这些发现支持将其作为标准治疗控制不佳的中重度疾病的附加疗法。 系统评价注册：https://www.crd.york.ac.uk/PROSPERO/view/CRD420251132811，标识符 CRD420251132811。
完整摘要 · 英文原文
OBJECTIVES: This meta-analysis aimed to synthesize data from English-language randomized controlled trials (RCTs) to evaluate the efficacy and safety of omalizumab in patients aged 6-20 years with allergic asthma, with specific attention to age-stratified outcomes where feasible. METHODS: Conducted in accordance with the PRISMA 2020 guidelines, this review followed a pre-registered protocol (PROSPERO: CRD420251132811). Eligible studies were English-language RCTs involving predominantly pediatric and adolescent patients with allergic asthma; one included trial enrolled participants up to 20 years of age, but only 15 participants (0.8% of the total sample) were aged 18-20 years, and all remaining participants were <18 years. Studies compared omalizumab with control treatment (placebo or standard therapy). Efficacy outcomes included asthma exacerbations, forced expiratory volume in 1 s (FEV1), and Childhood Asthma Control Test (C-ACT) scores. Safety outcomes encompassed treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and asthma-related hospitalizations. Data were pooled using random-effects models where appropriate, with heterogeneity assessed via I2 statistics. RESULTS: Six RCTs involving 1,835 participants (omalizumab: 1,134; control: 701) aged 6-20 years were included. Omalizumab significantly reduced asthma exacerbations (relative risk RR = 0.66, 95% CI: 0.55-0.79; I2 = 51.6%), SAEs (RR = 0.56, 95% CI: 0.35-0.90; I2 = 43.1%), and hospitalizations (RR = 0.35, 95% CI: 0.22-0.56; I2 = 0.0%). No significant difference was found in TEAEs (RR = 0.96, 95% CI: 0.92-1.01; I2 = 1.5%). Evidence regarding lung function and asthma control was limited and could not be quantitatively pooled. CONCLUSION: Omalizumab is effective in reducing exacerbations, SAEs, and hospitalizations in pediatric and adolescent allergic asthma, with a favorable safety profile. Evidence regarding lung function and asthma control remains limited and should be interpreted cautiously. These findings support its use as an add-on therapy for moderate-to-severe disease inadequately controlled by standard treatments. SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/PROSPERO/view/CRD420251132811, identifer CRD420251132811.