Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor
Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor
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DOI：
10.1016/j.jcf.2017.06.002
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发表时间：
2018-01-01
影响因子：
5.2
通讯作者：
Wagener, Jeffrey S.
中科院分区：
文献类型：
--
作者：
Flume, Patrick A.;Wainwright, Claire E.;Wagener, Jeffrey S.
背景：肺急性加重（PEx）与肺功能急性丧失相关，治疗后往往无法恢复。我们调查肺功能恢复后PEx ivacaftor和安慰剂治疗的subjects.Methods：短期和长期肺功能恢复后PEx数据进行了总结，从安慰剂对照试验，在161囊性纤维化患者12岁的G551 D-CFTR突变（NCT 00909532）。短期恢复在治疗后2至8周进行测量，长期恢复在研究结束时进行测定，两者均与PEx前测量的基线值进行比较。（33.7% vs. 56.4%; P = 0.004），并且具有较低的PEx校正发生率（0.589 vs. 1.382; P < 0.001）。PEx随后在1 s内预测用力呼气量百分比完全短期恢复的比例相似（ivacaftor vs.安慰剂，57.1% vs. 53.7），以及长期恢复的患者比例（46.4% vs. 47.7%）。与安慰剂相比，依伐卡托治疗降低了PEx的频率，但没有改善PEx后肺功能完全恢复的速率。(C)2017年欧洲囊性纤维化协会。Elsevier B. V.出版，保留所有权利。
Background: Pulmonary exacerbations (PEx) are associated with acute loss of lung function that is often not recovered after treatment. We investigated lung function recovery following PEx for ivacaftor- and placebo-treated subjects.Methods: Short- and long-term pulmonary function recovery data after PEx were summarized from a placebo-controlled trial in 161 cystic fibrosis patients 12 years old with the G551D-CFTR mutation (NCT00909532). Short-term recovery was measured 2 to 8 weeks after treatment, and long-term recovery was determined at the end-of-study, both compared with baseline measured just prior to the PEx.Results: Fewer patients receiving ivacaftor experienced a PEx than patients receiving placebo (33.7% vs. 56.4%; P = 0.004) and had a lower adjusted incidence rate of PEx (0.589 vs. 1.382; P < 0.001). The proportion of PEx followed by full short-term recovery of percent predicted forced expiratory volume in 1 s was similar (ivacaftor vs. placebo, 57.1% vs. 53.7), as was the proportion of patients having long-term recovery (46.4% vs. 47.7%).Conclusions: Ivacaftor treatment reduces the frequency of PEx but does not improve on the rate of complete lung function recovery after PEx when compared with placebo. (C) 2017 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.