The PROSPECT study, a post-approval observational study in the U.S., showed no significant changes in lung function as measured by spirometry with clinical initiation of lumacaftor/ivacaftor. A sub-study within the PROSPECT study assessed the lung clearance index (LCI), as measured by multiple breath washout (MBW), a measure of lung function demonstrated to be sensitive among people with normal spirometry. Participants performed MBW prior to clinically initiating lumacaftor/ivacaftor therapy and for one year of follow-up. Similar to the whole PROSPECT study, this sub-study cohort (N = 49) had no significant absolute or relative changes in FEV1% predicted at any time point. LCI, however, decreased (improved) by 0.81 units or 5.3% (95% CI -9.7, -0.9%) at 1 month, 0.77 units or 5.9% at 3 months, 0.67 units or 5.9% at 6 months, and 0.55 units or 4.3% at 12 months. These results demonstrate the utility of the LCI in assessing treatment effects of relatively modest size in a heterogenous study population.

中文翻译：

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接受 lumacaftor/ivacaftor 治疗的 F508del/F508del 患者 LCI 的变化：来自前景研究的结果

PROSPECT 研究是一项在美国进行的批准后观察性研究，通过肺活量计测量，临床开始使用 lumacaftor/ivacaftor 时，肺功能没有显着变化。PROSPECT 研究中的一项子研究评估了肺清除指数 (LCI)，这是通过多次呼吸冲洗 (MBW) 测量的，这是一种肺功能测量，在肺功能正常的人中被证明是敏感的。参与者在临床开始 lumacaftor/ivacaftor 治疗之前进行了 MBW，并进行了一年的随访。与整个 PROSPECT 研究类似，该子研究队列 (N = 49) 在任何时间点预测的 FEV1% 没有显着的绝对或相对变化。然而，LCI 在 1 个月时下降（改善）0.81 个单位或 5.3%（95% CI -9.7，-0.9%），在 3 个月时下降 0.77 个单位或 5.9%，在 6 个月时下降（0.67 个单位或 5.9%，以及 0.55 个单位）或 4。12 个月时为 3%。这些结果证明了 LCI 在评估异质研究人群中相对适中的治疗效果方面的效用。