Jang Jae-Hyuk, Park ChulHyoung, Kim Chungsoo, Lee Youngsoo, Lee Eunyoung, Park Rae Woong, Park Hae-Sim
Department of Allergy & Clinical Immunology, Ajou University School of Medicine, Suwon, Korea.
Department of Biomedical Informatics, Ajou University School of Medicine, Suwon, Korea.
Yonsei Med J. 2025 Sep;66(9):545-555. doi: 10.3349/ymj.2024.0320.
Omalizumab improves clinical outcomes for patients with severe asthma (SA), but its long-term effectiveness and potential biomarkers for predicting patient response require further investigation. This study aimed to evaluate the real-world effectiveness of omalizumab in treating SA and to identify potential biomarkers for predicting a favorable treatment response.
Clinical outcomes were compared between asthma patients receiving omalizumab (omalizumab group) and those on inhaled corticosteroid with long-acting beta-agonist (ICS-LABA) alone (ICS-LABA group). Propensity score matching and Cox proportional hazards model were used to calculate hazard ratios (HRs). Study outcomes included severe asthma exacerbation (SAE), incompletely controlled asthma, intravenous (IV) corticosteroid use, and asthma-related hospitalization. Incompletely controlled asthma was defined by blood eosinophil counts ≥150 cells/µL, fractional exhaled nitric oxide (FeNO) ≥25 ppb, forced expiratory volume in one second (FEV1%) <80%, or SAE occurrence.
The omalizumab group had significantly lower risks of SAE (HR 0.17, =0.03), incompletely controlled asthma (HR 0.56, =0.04), IV corticosteroid treatment (HR 0.38, =0.02), and asthma-related hospitalization (HR 0.27, =0.05). Blood eosinophil count stayed lower in the omalizumab group. FEV1% was higher with the omalizumab group, while blood neutrophil count, FeNO, and serum total IgE showed no differences. Furthermore, subgroup analysis showed patients with treatment-favorable response (>50% reduction in systemic corticosteroid dose) exhibited decreased blood neutrophil counts but increased FEV1% and serum total IgE levels compared with the treatment-unfavorable group.
Omalizumab treatment effectively reduces SAE and improves lung function and asthma control. Blood neutrophil counts and serum total IgE may be potential biomarkers for predicting favorable responses to omalizumab treatment.
奥马珠单抗可改善重度哮喘（SA）患者的临床结局，但其长期有效性以及预测患者反应的潜在生物标志物仍需进一步研究。本研究旨在评估奥马珠单抗治疗SA的实际疗效，并确定预测良好治疗反应的潜在生物标志物。
比较接受奥马珠单抗治疗的哮喘患者（奥马珠单抗组）与仅接受吸入性糖皮质激素联合长效β受体激动剂（ICS-LABA）治疗的患者（ICS-LABA组）的临床结局。采用倾向评分匹配和Cox比例风险模型计算风险比（HRs）。研究结局包括重度哮喘加重（SAE）、哮喘控制不佳、静脉注射（IV）糖皮质激素使用以及与哮喘相关的住院治疗。哮喘控制不佳的定义为血嗜酸性粒细胞计数≥150个细胞/微升、呼出一氧化氮分数（FeNO）≥25 ppb、一秒用力呼气容积（FEV1%）<80%或发生SAE。
奥马珠单抗组发生SAE（HR 0.17，P = 0.03）、哮喘控制不佳（HR 0.56，P = 0.04）、IV糖皮质激素治疗（HR 0.38，P = 0.02）以及与哮喘相关住院治疗（HR 0.27，P = 0.05）的风险显著更低。奥马珠单抗组的血嗜酸性粒细胞计数持续较低。奥马珠单抗组的FEV1%更高，而血中性粒细胞计数、FeNO和血清总IgE无差异。此外，亚组分析显示，与治疗反应不佳组相比，治疗反应良好（全身糖皮质激素剂量降低>50%）的患者血中性粒细胞计数降低，但FEV1%和血清总IgE水平升高。
奥马珠单抗治疗可有效降低SAE并改善肺功能和哮喘控制。血中性粒细胞计数和血清总IgE可能是预测对奥马珠单抗治疗有良好反应的潜在生物标志物。
