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目的:探讨奥马珠单抗在中国真实世界临床实践中治疗中重度哮喘的疗效、安全性及管理特点。方法:回顾性分析2018年3月至2020年4月在广州医科大学附属第一manbet官网登录 接受奥马珠单抗治疗至少4个月的中重度哮喘患者79例，男30例，女49例，年龄14~76岁，中位年龄50岁。比较患者治疗前后的临床表现、血嗜酸性粒细胞计数、呼出气一氧化氮（FeNO）、肺功能、口服糖皮质激素剂量及不良反应等。两两前后比较采用配对 t检验或非参数配对Wilcoxon分析，组间比较用非参数独立样本Mann-Whitney分析，计数资料组间比较采用卡方检验或Fisher检验分析。 结果:奥马珠单抗治疗4个月后，哮喘患者哮喘控制评分（ACT）从17.0（13.0，19.0）分升至20.0（18.0，24.0）分（ P<0.001）；急性发作次数从1.0（0，1.0）次/4个月减至0（0，1.0）次/4个月（ P<0.001）；呼气峰流速（PEF）变异率从16.5（13.8，27.3）%降低至10.4（6.0，16.2）%（ P<0.001）；PEF占预计值%从71.7（51.4，91.6）%升高至87.5（65.2，105.5）%（ P<0.001）；FEV 1占预计值%从73.6（53.9，90.8）%升高至80.6（68.7，91.8）%（ P=0.007）；口服糖皮质激素（OCS）维持剂量从12.0（10.0，20.0）mg/d减至5.0（0，17.5）mg/d（ P=0.001）。治疗总体应答率达74.7%，其中过敏性哮喘治疗应答率（77.3%）较非过敏性哮喘（25.0%）高（ P=0.019）。5例完成1年期治疗资料显示，治疗1年后ACT评分、急性发作次数、PEF占预计值%、PEF变异率及OCS维持剂量较治疗前仍有改善。共有3例（3.8%）患者共发生不良反应3次（0.6%）。分层分析显示，奥马珠单抗治疗4个月后，达到推荐治疗剂量（足剂量）组ACT评分改善程度及PEF变异率下降程度［3.0（1.0，8.0）分，6.5（3.5，15.8）%］均较未达到推荐治疗剂量（不足剂量）组［1.0（-0.3，3.0）分，2.9（1.5，5.0）%］明显，差异均有统计学意义（ P<0.05），且足剂量组治疗应答率（80.0%）较不足剂量组（50.0%）高（ P=0.019）。治疗有效但治疗时间不足1年停药的主要原因为经济因素（70.3%），其次是患者认为症状已改善不需继续用药（21.9%）及疗效不及预期（7.8%）。 结论:应用奥马珠单抗治疗中重度过敏性哮喘，初步观察具有良好疗效，不良反应少。足剂量组治疗应答率更高。经济因素是奥马珠单抗治疗有效但治疗时间不足1年停药的主要原因。

Objective:To observe the effectiveness, safety and management of omalizumab therapy for moderate to severe asthma in real-world clinical practice in China.Methods:This retrospective analysis involved 79 patients with moderate to severe asthma who received omalizumab therapy for at least 4 months in the First Affiliated Hospital of Guangzhou Medical University from March 2018 to April 2020. All participants were between 14 to 76 years old(median 50 years),including 30 males and 49 females. Data regarding the patients′ clinical manifestations, eosinophil count, fractional exhaled nitric oxide (FeNO), lung function, oral corticosteroid dosage, and adverse reactions were collected before and after treatment. Paired t-test or non-parametric paired Wilcoxon analysis was used for pairwise comparison, Mann Whitney analysis for inter-group comparison, and Chi square test or Fisher test for inter-group comparison of count data. Results:The following changes were noted after 4 months of omalizumab thearpy. The patients′ Asthma Control Test (ACT) scores increased from 17.0 (13.0-19.0) to 20.0 (18.0-24.0) points ( P<0.001). The frequency of acute exacerbations(AE) decreased from 1.0 (0-1.0) to 0 (0-1.0) episodes every 4 months ( P<0.001). The variation rate of the peak expiratory flow (PEF) decreased from 16.5 (13.8-27.3)% to 10.4 (6.0-16.2)% ( P<0.001). The percent predicted value of PEF (PEFpred%) increased from 71.7 (51.4-91.6)% to 87.5 (65.2-105.5)% ( P<0.001). The percent predicted value of the forced expiratory volume in 1 second(FEV 1%pred) increased from 73.6 (53.9-90.8)% to 80.6 (68.7-91.8)% ( P=0.007). The maintenance dose of oral corticosteroids (OCS) decreased from 12.0 (10.0-20.0) to 5.0 (0-17.5) mg/day ( P=0.001). After 4 months of treatment, the response rate of the 79 patients with asthma was 74.7%. The response rate of patients with allergic asthma (77.3%) was higher than that of patients with non-allergic asthma (25.0%) ( P=0.019). Among 5 patients who completed 1 year of treatment, the ACT score, frequency of AE, PEFpred%, variation rate of PEF and OCS maintenance dose were still improved after 1 year of treatment. Adverse reactions occurred in 3 patients (3.8%), for a total of 3 (0.6%) times. Stratified analysis showed that after 4 months of treatment, the improvement in the ACT score and the decrease in the PEF variation rate among patients who reached the recommended treatment dose (full dose) [3.0 (1.0-8.0) points, 6.5 (3.5-15.8) %] were significantly higher than those among patients who did not reach the recommended treatment dose (insufficient dose) [1.0 (-0.3-3.0) points, 2.9 (1.5-5.0) %] ( P<0.05). Additionally, the treatment response rate in patients with a sufficient dose (80.0%) was higher than that in patients with an insufficient dose (50.0%) ( P=0.019).The main factors associated with stopping treatment within 1 year despite a response to omalizumab was economic burden (70.3%), followed by satisfactory improvement by self-evaluation (21.9%) and less improvement in symptoms than expected (7.8%). Conclusion:Omalizumab was an effective treatment for moderate to severe allergic asthma with few adverse effects. The response rate was higher when the recommended injection dose was achieved. Financial difficulty was the main reason for stopping treatment within 1 year despite a good therapeutic response.