CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV₁ or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.

CFTR 功能是正常粘膜纤毛清除 (MCC) 和咳嗽辅助清除 (CC) 所必需的。Lumacaftor-ivacaftor 被批准用于携带两份 F508del-CFTR 的囊性纤维化 (CF) 患者。在这项在四个研究地点进行的观察性研究中，我们描述了 lumacaftor-ivacaftor 对粘膜纤毛和咳嗽清除的影响，并将其与治疗一个月后的其他临床和研究终点相关联。招收了 25 名青少年和成人。未观察到对全肺 MCC 的影响，但 CC 显着增加。_{该组的汗液氯化物改善了 18 mEq/L，表明 CFTR 活性适度恢复，但 FEV 1}没有明显变化或观察肺清除指数。我们推测 lumacaftor-ivacaftor 对 CFTR 功能的适度影响不足以产生 MCC 的改善。