Niven R, Chung K F, Panahloo Z, Blogg M, Ayre G
North West Lung Centre, Wythenshawe Hospital, Southmoor Road, Manchester, M23 9LT, UK.
Respir Med. 2008 Oct;102(10):1371-8. doi: 10.1016/j.rmed.2008.06.002. Epub 2008 Jul 26.
In a 1-year, randomized, open-label study in patients with moderate-to-severe allergic (immunoglobulin E (IgE)-mediated) asthma, adding omalizumab to best standard care (BSC) significantly improved efficacy outcomes compared with BSC alone (control). We assessed the efficacy of omalizumab in the subgroup of patients with inadequately controlled severe persistent allergic asthma despite high-dose inhaled corticosteroids (ICS) plus a long-acting beta(2)-agonist (LABA), which reflects the European Union (EU) label population.
Efficacy outcomes included annual asthma exacerbation rate, annual asthma deterioration-related incident (ADRI) rate, % predicted forced expiratory volume in 1 s (FEV(1)), asthma symptoms (Wasserfallen score) and quality of life (Mini Asthma Quality of Life Questionnaire (Mini-AQLQ)), which were compared in the omalizumab and control groups. Outcomes were also determined for omalizumab-treated patients judged to have responded to therapy (> or = 0.5-point improvement in Mini-AQLQ overall score at 27 weeks).
In total, 164 patients (omalizumab, n=115; control, n=49) were receiving high-dose ICS plus a LABA. Annual asthma exacerbation rate was significantly reduced by 59% in the omalizumab group vs. control (1.26 vs. 3.06; P<0.001). ADRI rate was significantly reduced by 40% in the omalizumab group compared with control (5.61 vs. 9.40; P<0.05). Significant improvements were also seen in % predicted FEV(1) (71% vs. 60%; P<0.001), change from baseline in asthma symptom scores (-6.7 vs. 0.5; P<0.05) and Mini-AQLQ overall score (1.32 vs. 0.17; P<0.001). In omalizumab-treated patients, 71/102 (70%) were judged to have responded to therapy. In these Mini-AQLQ-assessed responders, exacerbation rate was reduced by 64% vs. control (1.12 vs. 3.06; P<0.001), ADRI rate was reduced by 50% vs. control (4.71 vs. 9.40; P<0.01). Percent predicted FEV(1) (73% vs. 60%; P<0.001), change from baseline in asthma symptom scores (-8.1 vs. 0.5; P<0.001) and Mini-AQLQ overall score (1.81 vs. 0.17; P<0.001) were also further significantly improved vs. control.
Adding omalizumab to BSC is efficacious in patients with inadequately controlled severe persistent allergic asthma despite high-dose ICS plus a LABA (EU label population), with further efficacy observed in patients judged to have responded to therapy which may more accurately illustrate the actual benefit of omalizumab therapy in clinical practice. The naturalistic setting of this study confirms the benefits observed in double-blind randomized clinical trials.
在一项针对中重度过敏性（免疫球蛋白E（IgE）介导）哮喘患者的为期1年的随机、开放标签研究中，与单纯最佳标准治疗（BSC，对照）相比，在BSC基础上加用奥马珠单抗可显著改善疗效指标。我们评估了奥马珠单抗在尽管使用高剂量吸入性糖皮质激素（ICS）加长效β2受体激动剂（LABA）但仍控制不佳的重度持续性过敏性哮喘患者亚组中的疗效，该亚组反映了欧盟标签人群。
疗效指标包括年度哮喘加重率、年度哮喘恶化相关事件（ADRI）率、1秒用力呼气容积（FEV1）预测值百分比、哮喘症状（Wasserfallen评分）和生活质量（哮喘生活质量小问卷（Mini-AQLQ）），对奥马珠单抗组和对照组进行比较。还对判断为对治疗有反应（27周时Mini-AQLQ总分改善≥0.5分）的奥马珠单抗治疗患者的结局进行了测定。
共有164例患者（奥马珠单抗组115例，对照组49例）接受高剂量ICS加LABA治疗。奥马珠单抗组的年度哮喘加重率较对照组显著降低59%（1.26对3.06；P<0.001）。与对照组相比，奥马珠单抗组的ADRI率显著降低40%（5.61对9.40；P<0.05）。FEV1预测值百分比（71%对60%；P<0.001）、哮喘症状评分较基线的变化（-6.7对0.5；P<0.05）和Mini-AQLQ总分（1.32对0.17；P<0.001）也有显著改善。在接受奥马珠单抗治疗的患者中，102例中有71例（70%）被判断为对治疗有反应。在这些经Mini-AQLQ评估有反应的患者中，加重率较对照组降低64%（1.12对3.06；P<0.001），ADRI率较对照组降低50%（4.71对9.40；P<0.01）。与对照组相比，FEV1预测值百分比（73%对60%；P<0.001）、哮喘症状评分较基线的变化（-8.1对0.5；P<0.001）和Mini-AQLQ总分（1.81对0.17；P<0.001）也进一步显著改善。
在尽管使用高剂量ICS加LABA但仍控制不佳的重度持续性过敏性哮喘患者（欧盟标签人群）中，在BSC基础上加用奥马珠单抗是有效的，在判断为对治疗有反应的患者中观察到了进一步的疗效，这可能更准确地说明了奥马珠单抗治疗在临床实践中的实际益处。本研究的自然主义背景证实了在双盲随机临床试验中观察到的益处。
