Department of Internal Medicine, University of Iowa, Iowa City, Iowa, USA.
Department of Biostatistics, University of Iowa, Iowa City, Iowa, USA.
Clin Infect Dis. 2022 Sep 30;75(7):1115-1122. doi: 10.1093/cid/ciac117.
People with cystic fibrosis (CF) routinely suffer from recurrent sinopulmonary infections. Such infections require frequent courses of antimicrobials and often involve multidrug-resistant organisms. The goal of this study was to identify real-world evidence for the effectiveness of elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) in decreasing infection-related visits and antimicrobial use in people with CF.
Using IBM MarketScan data, we identified 389 enrollees with CF who began taking ELX/TEZ/IVA before 1 December 2019 and were enrolled from 1 July 2019 to 14 March 2020. We also identified a comparison population who did not begin ELX/TEZ/IVA during the study period. We compared the following outcomes in the 15 weeks before and after medication initiation: total healthcare visits, inpatient visits, infection-related visits, and antimicrobial prescriptions. We analyzed outcomes using both a case-crossover analysis and a difference-in-differences analysis, to control for underlying trends.
For the case-crossover analysis, ELX/TEZ/IVA initiation was associated with the following changes over a 15-week period: change in overall healthcare visit dates, -2.5 (95% confidence interval, -3.31 to -1.7); change in inpatient admissions, -0.16 (-.22 to -.10); change in infection-related visit dates, -0.62 (-.93 to -.31); and change in antibiotic prescriptions, -0.78 (-1.03 to -.54). Results from the difference-in-differences approach were similar.
We show a rapid reduction in infection-related visits and antimicrobial use among people with CF after starting a therapy that was not explicitly designed to treat infections. Currently, there are >30 000 people living with CF in the United States alone. Given that this therapy is effective for approximately 90% of people with CF, the impact on respiratory infections and antimicrobial use may be substantial.
囊性纤维化(CF)患者经常遭受反复的鼻窦肺部感染。此类感染需要频繁使用抗生素治疗,并且常常涉及耐多药病原体。本研究的目的是确定真实世界证据,证明依伐卡托与泰比曲肽和艾美拉唑(ELX/TEZ/IVA)在减少 CF 患者感染相关就诊和抗生素使用方面的有效性。
我们使用 IBM MarketScan 数据,确定了 389 名在 2019 年 12 月 1 日前开始服用 ELX/TEZ/IVA 的 CF 患者,并在 2019 年 7 月 1 日至 2020 年 3 月 14 日期间入组。我们还确定了一个在研究期间未开始服用 ELX/TEZ/IVA 的对照组。我们比较了药物起始前 15 周和后 15 周的以下结果:总医疗就诊次数、住院就诊次数、感染相关就诊次数和抗生素处方。我们使用病例交叉分析和差异分析来分析结果,以控制潜在趋势。
对于病例交叉分析,ELX/TEZ/IVA 起始治疗与 15 周内的以下变化相关:总医疗就诊日期的变化,-2.5(95%置信区间,-3.31 至-1.7);住院入院的变化,-0.16(-0.22 至-0.10);感染相关就诊日期的变化,-0.62(-0.93 至-0.31);抗生素处方的变化,-0.78(-1.03 至-0.54)。差异分析的结果相似。
我们发现,开始治疗后 CF 患者的感染相关就诊和抗生素使用迅速减少,而该治疗方案并非专门用于治疗感染。目前,仅在美国就有超过 30000 人患有 CF。鉴于该疗法对约 90%的 CF 患者有效,对呼吸道感染和抗生素使用的影响可能是巨大的。