Corren Jonathan, Kavati Abhishek, Ortiz Benjamin, Colby Jennifer A, Ruiz Kimberly, Maiese Brett A, Cadarette Sarah M, Panettieri Reynold A
Allergy Asthma Proc. 2017 Jul 1;38(4):250-263. doi: 10.2500/aap.2017.38.4067. Epub 2017 Jun 19.
There are limited pediatric data about the use of omalizumab, especially the effectiveness and safety of omalizumab in the real-world management of allergic asthma.
The objective of this study was to summarize the safety and efficacy of omalizumab in both randomized clinical trials (RCT) used for U.S. Food and Drug Administration registration and real-world studies (RWS) based on clinical care of children with moderate-to-severe asthma.
Studies that evaluated omalizumab use in patients <18 years old and with asthma, published between January 2003 and October 2016, were retrieved from medical literature data bases. Assessed outcomes included the following: exacerbation rates, spirometric indices, changes in asthma medication use, asthma control, patient-reported outcomes, and health care resource utilization.
A total of five RWS were identified; outcomes reported were compared with three omalizumab RCTs. Overall, the mean rate of annual exacerbations was significantly lower after 6 months to 2 years of treatment with omalizumab in both RCTs and RWS. In two RCTs and three RWS, inhaled corticosteroid use was significantly reduced in patients who used omalizumab. Similar reductions in the use of rescue medication were also observed in the RCTs and RWS on omalizumab. Real-world evidence demonstrated improvement in forced expiratory volume in the first second of expiration (% predicted) in patients treated with omalizumab as well as significant improvement in the level of asthma control observed over 1 year. There also was evidence that omalizumab treatment reduced health care resource utilization, including fewer hospitalizations, emergency department visits, and unscheduled medical visits. Safety outcomes in all five RWS showed no new safety signals and demonstrated that omalizumab was well tolerated.
Overall, RCT evidence strongly supported omalizumab efficacy and safety as add-on treatment in children 6 to 11 years old with moderate-to-severe persistent allergic asthma. RWS data confirmed these findings in an extended patient population of children and adolescents that is more generalizable to the actual day-to-day management of these patients.
关于奥马珠单抗使用的儿科数据有限，尤其是其在过敏性哮喘实际治疗中的有效性和安全性。
本研究的目的是总结奥马珠单抗在美国食品药品监督管理局注册用的随机临床试验（RCT）以及基于中重度哮喘患儿临床护理的真实世界研究（RWS）中的安全性和有效性。
从医学文献数据库中检索2003年1月至2016年10月间发表的评估奥马珠单抗用于18岁以下哮喘患者的研究。评估的结果包括以下各项：急性加重率、肺功能指标、哮喘药物使用变化、哮喘控制情况、患者报告的结果以及卫生保健资源利用情况。
共确定了5项真实世界研究；将报告的结果与3项奥马珠单抗随机临床试验进行了比较。总体而言，在随机临床试验和真实世界研究中，使用奥马珠单抗治疗6个月至2年后，年急性加重的平均发生率均显著降低。在2项随机临床试验和3项真实世界研究中，使用奥马珠单抗的患者吸入糖皮质激素的使用显著减少。在奥马珠单抗的随机临床试验和真实世界研究中也观察到急救药物使用有类似减少。真实世界证据表明，接受奥马珠单抗治疗的患者第1秒用力呼气量（预测值%）有所改善，且在1年中观察到哮喘控制水平有显著改善。也有证据表明奥马珠单抗治疗减少了卫生保健资源利用，包括住院、急诊就诊和非计划医疗就诊次数减少。所有5项真实世界研究中的安全性结果均未显示新的安全信号，并表明奥马珠单抗耐受性良好。
总体而言，随机临床试验证据有力支持奥马珠单抗作为6至11岁中重度持续性过敏性哮喘患儿附加治疗的有效性和安全性。真实世界研究数据在更广泛的儿童和青少年患者群体中证实了这些发现，这对于这些患者的实际日常管理更具普遍性。
