Sex Differences After Treatment With Ivacaftor in People With Cystic Fibrosis.

IF 9.5 1区 医学 Q1 CRITICAL CARE MEDICINE Chest Pub Date : 2024-11-01 Epub Date: 2024-06-11 DOI:10.1016/j.chest.2024.05.019
Melanie Holtrop, Sophia Cosmich, MinJae Lee, Ashley Keller, Raksha Jain
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Abstract

Background: Historically, studies show that female patients with cystic fibrosis (CF) have worse pulmonary outcomes than male patients, including decreased life expectancy. It is unknown whether this disparity persists in the new era of highly effective modulator therapies. Ivacaftor has been available in the United States for > 10 years, allowing for the opportunity to understand the impact this therapy may have on sex disparities in CF. We hypothesized that female patients will continue to show worse outcomes because we suspect that the disparity is not driven solely by ion channel dysfunction.

Research question: Does a difference in outcomes between male and female patients persist after the initiation of ivacaftor in people with CF?

Study design and methods: We conducted a retrospective cohort study using the CF Foundation Patient Registry comparing changes in pulmonary exacerbation rate, lung function (FEV1 % predicted), and presence of Pseudomonas aeruginosa among male patients vs female patients before and after initiation of treatment with the highly effective modulator ivacaftor.

Results: The cohort comprised 1,900 people with CF who were treated with ivacaftor between 2010 and 2017; 928 patients (48.84%) were male and 972 patients (51.16%) were female with a mean age of 33.09 years. Male patients showed a significant decrease in pulmonary exacerbations after ivacaftor treatment (from 0.38 to 0.34; adjusted rate ratio, 0.89; P = .028), whereas female patients did not (from 0.48 to 0.45; adjusted rate ratio, 0.95; P = .174). FEV1 % predicted similarly decreased in both male and female patients before vs after ivacaftor treatment. P aeruginosa prevalence decreased to a similar extent in both male and female patients after ivacaftor treatment.

Interpretation: Our findings demonstrate that sex disparities in CF persist in those treated with ivacaftor because of differences in pulmonary exacerbations. More research is needed to determine the specific pathophysiologic drivers of this disparity.

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囊性纤维化患者接受伊伐卡夫托治疗后仍存在性别差异。
背景:历史研究表明,囊性纤维化(CF)女性患者的肺部治疗效果比男性患者差,包括预期寿命缩短。在使用高效调节剂疗法(HEMTs)的新时代,这种差异是否依然存在尚不得而知。伊伐卡夫托(Ivacaftor)在美国上市已超过十年,因此我们有机会了解这种疗法对 CF 性别差异的影响。我们假设女性的预后将继续恶化,因为我们怀疑这种差异并不完全是由离子通道功能障碍造成的:研究设计和方法:我们利用CF基金会患者登记处(CFFPR)进行了一项回顾性队列研究,比较了开始使用高效调节剂伊伐卡夫托治疗前后男性与女性在肺部恶化率、肺功能(ppFEV1)和铜绿假单胞菌存在情况方面的变化:我们的队列包括2010-2017年间接受伊伐卡夫托治疗的1900名CF患者,其中男性928人(48.84%),女性972人(51.16%),平均年龄33.09岁。ivacaftor治疗后,男性的肺部恶化率明显降低(从0.38降至0.34,调整后比率为0.89,p = 0.028),而女性则没有明显降低(从0.48降至0.45,调整后比率为0.95,p = 0.174)。ivacaftor治疗后,男性和女性铜绿假单胞菌感染率的下降程度相似:我们的研究结果表明,在接受伊伐卡夫托治疗的患者中,由于肺部恶化的不同,CF患者的性别差异依然存在。要确定造成这种差异的具体病理生理学因素,还需要进行更多的研究。
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来源期刊
Chest
Chest 医学-呼吸系统
CiteScore
13.70
自引率
3.10%
发文量
3369
审稿时长
15 days
期刊介绍: At CHEST, our mission is to revolutionize patient care through the collaboration of multidisciplinary clinicians in the fields of pulmonary, critical care, and sleep medicine. We achieve this by publishing cutting-edge clinical research that addresses current challenges and brings forth future advancements. To enhance understanding in a rapidly evolving field, CHEST also features review articles, commentaries, and facilitates discussions on emerging controversies. We place great emphasis on scientific rigor, employing a rigorous peer review process, and ensuring all accepted content is published online within two weeks.
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