贝那利珠单抗在东京哮喘研究（TOAST）中的有效性：一项现实世界的前瞻性介入试验
2025/01/26
摘要
背景：生物制剂在严重哮喘的治疗中是不可或缺的。由于抗IL-5受体抗体贝那利珠单抗在日本的有效性仍然难以捉摸，因此本研究旨在评估其在日本严重哮喘患者中的实际有效性。
方法：这项前瞻性，介入性，单臂临床试验于2020年9月至2022年7月在日本的十个设施中进行。招募患有严重嗜酸性粒细胞哮喘（外周血嗜酸性粒细胞计数≥150个细胞/μl）的成年患者，并用贝那利珠单抗治疗。主要终点是ACQ-5评分从基线到第24周的变化。
结果：103例患者中，98例（平均年龄62.1岁，女性55.1%，常规口服皮质类固醇治疗20.4%）被纳入分析。从基线到第24周，贝那利珠单抗显着改善ACQ-5（-0.67，95%CI：-0.94至-0.39）和AQLQ（0.71，95%CI:0.46至0.96）评分，FEV1增加（87 ml，95%CI:15-159 ml）。维持OCS剂量和OCS使用者百分比分别从13.9 mg/天降至6.0 mg/天和20.4%降至9.2%。多变量分析确定基线血嗜酸性粒细胞计数（≥400细胞/μl）和呼出一氧化氮分数（≥22 ppb）是贝那利珠单抗治疗反应的独立预测因子。贝那利珠单抗治疗因非严重不良事件和患者选择分别在四名和三名患者中停止。这项前瞻性，介入性，单臂临床试验于2020年9月至2022年7月在日本的十个设施中进行。招募患有严重嗜酸性粒细胞哮喘（外周血嗜酸性粒细胞计数≥150个细胞/μl）的成年患者，并用贝那利珠单抗治疗。主要终点是ACQ-5评分从基线到第24周的变化。
结论：在日本的现实环境中，用贝那利珠单抗治疗的严重嗜酸性粒细胞性哮喘患者在哮喘控制，生活质量和呼吸功能方面表现出显着改善，OCS使用减少。
Effectiveness of benralizumab in the Tokyo Asthma Study (TOAST): A real-world prospective interventional trial.
Katsunori, Masaki; Maho, Suzukawa;
Abstrast
Background: Biologics are integral in the management of severe asthma. As the effectiveness of the anti-IL-5 receptor antibody benralizumab in Japan remains elusive, this study aimed to assess its real-world effectiveness in Japanese patients with severe asthma.
Methods: This prospective, interventional, single-arm clinical trial was conducted across ten facilities in Japan between September 2020 and July 2022. Adult patients with severe eosinophilic asthma (peripheral blood eosinophil count ≥150 cells/μl) were enrolled and treated with benralizumab. The primary endpoint was the change in ACQ-5 score from baseline to week 24.
Results：Of 103 patients, 98 (mean age: 62.1 years, women: 55.1 %, regular oral corticosteroids [OCS] treatment: 20.4 %) were included in the analysis. From baseline to week 24, benralizumab significantly improved ACQ-5 (-0.67, 95 % CI: -0.94 to -0.39) and AQLQ (0.71, 95 % CI: 0.46 to 0.96) scores with an increase in FEV1 (87 ml, 95 % CI: 15-159 ml). The maintenance OCS dose and the percentage of OCS users decreased from 13.9 mg/day to 6.0 mg/day and from 20.4 % to 9.2 %, respectively. Multivariable analysis identified baseline blood eosinophil count (≥400 cells/μl) and fractional exhaled nitric oxide (≥22 ppb) as independent predictors of therapeutic response to benralizumab. Benralizumab treatment was discontinued due to nonserious adverse events and patient choice in four and three patients, respectively.
Conclusions: In a real-world setting in Japan, patients with severe eosinophilic asthma treated with benralizumab demonstrated substantial improvements in asthma control, quality of life, and respiratory function with reduced OCS usage.
背景：生物制剂在严重哮喘的治疗中是不可或缺的。由于抗IL-5受体抗体贝那利珠单抗在日本的有效性仍然难以捉摸，因此本研究旨在评估其在日本严重哮喘患者中的实际有效性。
方法：这项前瞻性，介入性，单臂临床试验于2020年9月至2022年7月在日本的十个设施中进行。招募患有严重嗜酸性粒细胞哮喘（外周血嗜酸性粒细胞计数≥150个细胞/μl）的成年患者，并用贝那利珠单抗治疗。主要终点是ACQ-5评分从基线到第24周的变化。
结果：103例患者中，98例（平均年龄62.1岁，女性55.1%，常规口服皮质类固醇治疗20.4%）被纳入分析。从基线到第24周，贝那利珠单抗显着改善ACQ-5（-0.67，95%CI：-0.94至-0.39）和AQLQ（0.71，95%CI:0.46至0.96）评分，FEV1增加（87 ml，95%CI:15-159 ml）。维持OCS剂量和OCS使用者百分比分别从13.9 mg/天降至6.0 mg/天和20.4%降至9.2%。多变量分析确定基线血嗜酸性粒细胞计数（≥400细胞/μl）和呼出一氧化氮分数（≥22 ppb）是贝那利珠单抗治疗反应的独立预测因子。贝那利珠单抗治疗因非严重不良事件和患者选择分别在四名和三名患者中停止。这项前瞻性，介入性，单臂临床试验于2020年9月至2022年7月在日本的十个设施中进行。招募患有严重嗜酸性粒细胞哮喘（外周血嗜酸性粒细胞计数≥150个细胞/μl）的成年患者，并用贝那利珠单抗治疗。主要终点是ACQ-5评分从基线到第24周的变化。
结论：在日本的现实环境中，用贝那利珠单抗治疗的严重嗜酸性粒细胞性哮喘患者在哮喘控制，生活质量和呼吸功能方面表现出显着改善，OCS使用减少。
(中日友好医院呼吸与危重症医学科 万静萱 摘译 林江涛 审校)
(Allergol Int 2024 Dec 3;0(0). DOI: 10.1016/j.alit.2024.10.009.IF：4.806)
(Allergol Int 2024 Dec 3;0(0). DOI: 10.1016/j.alit.2024.10.009.IF：4.806)
Effectiveness of benralizumab in the Tokyo Asthma Study (TOAST): A real-world prospective interventional trial.
Katsunori, Masaki; Maho, Suzukawa;
Abstrast
Background: Biologics are integral in the management of severe asthma. As the effectiveness of the anti-IL-5 receptor antibody benralizumab in Japan remains elusive, this study aimed to assess its real-world effectiveness in Japanese patients with severe asthma.
Methods: This prospective, interventional, single-arm clinical trial was conducted across ten facilities in Japan between September 2020 and July 2022. Adult patients with severe eosinophilic asthma (peripheral blood eosinophil count ≥150 cells/μl) were enrolled and treated with benralizumab. The primary endpoint was the change in ACQ-5 score from baseline to week 24.
Results：Of 103 patients, 98 (mean age: 62.1 years, women: 55.1 %, regular oral corticosteroids [OCS] treatment: 20.4 %) were included in the analysis. From baseline to week 24, benralizumab significantly improved ACQ-5 (-0.67, 95 % CI: -0.94 to -0.39) and AQLQ (0.71, 95 % CI: 0.46 to 0.96) scores with an increase in FEV1 (87 ml, 95 % CI: 15-159 ml). The maintenance OCS dose and the percentage of OCS users decreased from 13.9 mg/day to 6.0 mg/day and from 20.4 % to 9.2 %, respectively. Multivariable analysis identified baseline blood eosinophil count (≥400 cells/μl) and fractional exhaled nitric oxide (≥22 ppb) as independent predictors of therapeutic response to benralizumab. Benralizumab treatment was discontinued due to nonserious adverse events and patient choice in four and three patients, respectively.
Conclusions: In a real-world setting in Japan, patients with severe eosinophilic asthma treated with benralizumab demonstrated substantial improvements in asthma control, quality of life, and respiratory function with reduced OCS usage.
上一篇：
Dupilumab对2型哮喘患者呼出一氧化氮、黏液栓和功能性呼吸成像的影响（VESTIGE）：一项随机、双盲、安慰剂对照
下一篇：
贝纳利珠单抗（ABRA）治疗哮喘和COPD的嗜酸性粒细胞加重：一项双盲、双模拟、有效安慰剂对照随机试验
