特泽利尤单抗在2型（T2）与非2型重症哮喘表型中的真实世界疗效：一项西班牙多中心队列研究
2026/06/30
摘要
研究目的：评估特泽利尤单抗（tezepelumab）在重症哮喘患者中的真实世界疗效，分析药物在不同2型（T2）炎症状态、不同既往生物制剂暴露史人群中的效果差异，并明确疗效预测因素。
研究方法：本研究为覆盖西班牙17家三级医院的多中心回顾性队列研究，从药房发药记录中筛选出符合欧洲呼吸学会/美国胸科学会（ERS/ATS）重症哮喘定义、初始启动特泽利尤单抗治疗的成人患者。研究终点包括：哮喘控制测试（ACT）评分、年化急性加重次数、口服糖皮质激素（OCS）暴露情况（维持用药比例、泼尼松日剂量>5mg占比、累积剂量）、肺功能指标（一秒用力呼气容积FEV1、FEV1占预计值百分比）、血嗜酸粒细胞计数、呼出气一氧化氮（FeNO）水平。采用线性回归分析评估ACT评分和急性加重变化的预测因子。
研究结果：307例初始用药患者中，共304例完成配对评估。平均ACT评分从13.6升至17.0，平均提升3.4分；年化急性加重次数从2.9次降至0.8次，47%的患者达到无急性加重状态。OCS维持用药比例从22.4%降至16.8%，泼尼松日剂量>5mg的占比从22.4%降至6.9%，降幅达70%；OCS累积中位剂量从420mg降至0mg，平均累积剂量从1575mg降至1020mg。平均FEV1从1850mL升至1930mL，FEV1占预计值百分比从70.4%升至73.7%，且基线FEV1占预计值<80%的患者肺功能改善幅度更大。12.3%的患者达到临床缓解；随访结束时，54.8%的患者血嗜酸粒细胞计数<150个/μL，74.6%的患者FeNO<25ppb。分析显示，既往存在生物制剂暴露史的患者，ACT评分改善幅度和急性加重减少幅度均更小。
研究结论：在专科诊疗场景下，特泽利尤单抗可显著改善不同表型重症哮喘的哮喘控制水平、减少急性发作、降低OCS暴露、轻度改善肺功能，并降低2型炎症生物标志物水平。探索性分析提示，年龄、性别、既往OCS用药史、T2炎症状态、既往生物制剂暴露史是特泽佩鲁单抗的疗效预测因素。未来仍需开展对照研究明确因果关系。
Real-World Effectiveness of Tezepelumab Across T2 and Non-T2 Severe Asthma Phenotypes: A Multicenter Spanish Cohort Study
Elena Villamañán, Carlos Carpio, Daniel Laorden, Javier Domínguez-Ortega, David Romero, Leticia De Las Vecillas, Alejandro Soto, Eva Villaroya, Belén Romero, Itsaso Losantos, Carmen Sobrino, Susana De Andrés, Alicia Herrero, Santiago Quirce, Rodolfo Álvarez-Sala; AsmaGrave-HULP study group, on behalf of the TezeRWD study group
Abstract
Objectives: To evaluate real-world effectiveness of tezepelumab in severe asthma, by type-2 (T2) status and prior biologic exposure, and response predictors.
Methods: Multicentre retrospective cohort in 17 Spanish tertiary hospitals. Adults with ERS/ATS-defined severe asthma initiating tezepelumab were identified from pharmacy dispensing records. Outcomes included Asthma Control Test (ACT), annualised exacerbations, oral corticosteroid (OCS) exposure (maintenance use, prednisone>5mg/day, cumulative dose), spirometry (FEV1; % predicted), blood eosinophils and fractional exhaled nitric oxide (FeNO). Linear regression assessed predictors of change in ACT and exacerbations.
Results: Of 307 initiators, 304 had paired assessments. Mean ACT rose from 13.6 to 17.0 (+3.4) and annualised exacerbations fell from 2.9 to 0.8; 47% were exacerbation-free. Maintenance OCS use decreased from 22.4% to 16.8%, and prednisone>5mg/day fell from 22.4% to 6.9% (70% reduction). Cumulative OCS dose declined from 1575 to 1020mg (median 420 to 0mg). Mean FEV1 increased from 1850 to 1930mL and % predicted from 70.4% to 73.7%, with larger gains at baseline FEV1<80% predicted. Clinical remission was achieved in 12.3% of patients. At follow-up, 54.8% had eosinophils<150cells/μL and 74.6% had FeNO<25ppb. Prior biologic exposure predicted smaller improvements in ACT and exacerbations.
Conclusions: In specialist care, tezepelumab was associated with meaningful improvements in asthma control, fewer exacerbations, reduced OCS exposure, modest lung function gains, and reductions in T2 biomarkers across phenotypes. Exploratory analyses identified age, sex, prior OCS use, T2 status and prior biologic exposure as response predictors. Future comparative studies are needed to confirm causality.
研究目的：评估特泽利尤单抗（tezepelumab）在重症哮喘患者中的真实世界疗效，分析药物在不同2型（T2）炎症状态、不同既往生物制剂暴露史人群中的效果差异，并明确疗效预测因素。
研究方法：本研究为覆盖西班牙17家三级医院的多中心回顾性队列研究，从药房发药记录中筛选出符合欧洲呼吸学会/美国胸科学会（ERS/ATS）重症哮喘定义、初始启动特泽利尤单抗治疗的成人患者。研究终点包括：哮喘控制测试（ACT）评分、年化急性加重次数、口服糖皮质激素（OCS）暴露情况（维持用药比例、泼尼松日剂量>5mg占比、累积剂量）、肺功能指标（一秒用力呼气容积FEV1、FEV1占预计值百分比）、血嗜酸粒细胞计数、呼出气一氧化氮（FeNO）水平。采用线性回归分析评估ACT评分和急性加重变化的预测因子。
研究结果：307例初始用药患者中，共304例完成配对评估。平均ACT评分从13.6升至17.0，平均提升3.4分；年化急性加重次数从2.9次降至0.8次，47%的患者达到无急性加重状态。OCS维持用药比例从22.4%降至16.8%，泼尼松日剂量>5mg的占比从22.4%降至6.9%，降幅达70%；OCS累积中位剂量从420mg降至0mg，平均累积剂量从1575mg降至1020mg。平均FEV1从1850mL升至1930mL，FEV1占预计值百分比从70.4%升至73.7%，且基线FEV1占预计值<80%的患者肺功能改善幅度更大。12.3%的患者达到临床缓解；随访结束时，54.8%的患者血嗜酸粒细胞计数<150个/μL，74.6%的患者FeNO<25ppb。分析显示，既往存在生物制剂暴露史的患者，ACT评分改善幅度和急性加重减少幅度均更小。
研究结论：在专科诊疗场景下，特泽利尤单抗可显著改善不同表型重症哮喘的哮喘控制水平、减少急性发作、降低OCS暴露、轻度改善肺功能，并降低2型炎症生物标志物水平。探索性分析提示，年龄、性别、既往OCS用药史、T2炎症状态、既往生物制剂暴露史是特泽佩鲁单抗的疗效预测因素。未来仍需开展对照研究明确因果关系。
（中日友好医院呼吸与危重症医学科 李红雯 摘译 林江涛 审校）
（Arch Bronconeumol. 2026 Jun 4:S0300-2896(26)00214-0. doi: 10.1016/j.arbres.2026.05.022.）
Real-World Effectiveness of Tezepelumab Across T2 and Non-T2 Severe Asthma Phenotypes: A Multicenter Spanish Cohort Study
Elena Villamañán, Carlos Carpio, Daniel Laorden, Javier Domínguez-Ortega, David Romero, Leticia De Las Vecillas, Alejandro Soto, Eva Villaroya, Belén Romero, Itsaso Losantos, Carmen Sobrino, Susana De Andrés, Alicia Herrero, Santiago Quirce, Rodolfo Álvarez-Sala; AsmaGrave-HULP study group, on behalf of the TezeRWD study group
Abstract
Objectives: To evaluate real-world effectiveness of tezepelumab in severe asthma, by type-2 (T2) status and prior biologic exposure, and response predictors.
Methods: Multicentre retrospective cohort in 17 Spanish tertiary hospitals. Adults with ERS/ATS-defined severe asthma initiating tezepelumab were identified from pharmacy dispensing records. Outcomes included Asthma Control Test (ACT), annualised exacerbations, oral corticosteroid (OCS) exposure (maintenance use, prednisone>5mg/day, cumulative dose), spirometry (FEV1; % predicted), blood eosinophils and fractional exhaled nitric oxide (FeNO). Linear regression assessed predictors of change in ACT and exacerbations.
Results: Of 307 initiators, 304 had paired assessments. Mean ACT rose from 13.6 to 17.0 (+3.4) and annualised exacerbations fell from 2.9 to 0.8; 47% were exacerbation-free. Maintenance OCS use decreased from 22.4% to 16.8%, and prednisone>5mg/day fell from 22.4% to 6.9% (70% reduction). Cumulative OCS dose declined from 1575 to 1020mg (median 420 to 0mg). Mean FEV1 increased from 1850 to 1930mL and % predicted from 70.4% to 73.7%, with larger gains at baseline FEV1<80% predicted. Clinical remission was achieved in 12.3% of patients. At follow-up, 54.8% had eosinophils<150cells/μL and 74.6% had FeNO<25ppb. Prior biologic exposure predicted smaller improvements in ACT and exacerbations.
Conclusions: In specialist care, tezepelumab was associated with meaningful improvements in asthma control, fewer exacerbations, reduced OCS exposure, modest lung function gains, and reductions in T2 biomarkers across phenotypes. Exploratory analyses identified age, sex, prior OCS use, T2 status and prior biologic exposure as response predictors. Future comparative studies are needed to confirm causality.
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欧洲重症哮喘及其缓解前景研究（SHARP）：基于欧洲重症哮喘登记处的多中心观察性研究见解
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