BACKGROUND Hypertonic saline (HS) is commonly prescribed for children with cystic fibrosis (CF) despite the absence of strong data indicating clinical efficacy in a population with mild lung disease. We hypothesized that HS treatment would result in a sustained improvement in mucociliary clearance (MCC) in children with CF who had minimal lung disease, thus providing evidence for a biologically relevant effect that also may be associated with clinical improvements. METHODS We performed a randomized, placebo controlled, double blind study of 6% versus 0.12% sodium chloride, delivered three-times daily with an eFlow nebulizer for 4 weeks. MCC was measured using gamma scintigraphy at baseline, 2-hours after the first study treatment, and ~12-hours after the final dose (at day 28). Spirometry, respiratory symptoms (CFQ-R), and safety were also assessed. RESULTS Study treatments were generally well tolerated and safe. HS (6% sodium chloride) resulted in a significant, sustained improvement from baseline in whole lung clearance after 4 weeks of therapy (p = 0.014), despite absence of a prolonged single-dose effect after the initial dose. This sustained change (12 hrs after prior dose) was significantly greater when compared to placebo (0.12% sodium chloride) treatment (p = 0.016). Improvements in spirometry with HS did not reach statistical significance but correlated with MCC changes. CONCLUSIONS The observed sustained improvement in MCC with HS suggests that this treatment may yield health benefits, even in relatively mildly affected children with CF. Highlighting this physiologic finding is important due to the lack of meaningful, validated endpoints in this population.

中文翻译：

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对患有轻度囊性纤维化肺病的儿童使用高渗盐水进行为期 4 周的试验：对粘液纤毛清除和临床结果的影响

背景 高渗盐水 (HS) 通常用于患有囊性纤维化 (CF) 的儿童，尽管缺乏强有力的数据表明其在轻度肺病人群中的临床疗效。我们假设 HS 治疗会持续改善患有轻度肺部疾病的 CF 儿童的黏液纤毛清除率 (MCC)，从而为生物学相关效应提供证据，该效应也可能与临床改善相关。方法 我们对 6% 与 0.12% 氯化钠进行了一项随机、安慰剂对照、双盲研究，使用 eFlow 雾化器每天给药 3 次，持续 4 周。在基线、第一次研究治疗后 2 小时和最后一次给药后约 12 小时（第 28 天）使用伽马闪烁扫描法测量 MCC。肺活量测定法，呼吸道症状 (CFQ-R)，并且还评估了安全性。结果 研究治疗通常具有良好的耐受性和安全性。HS（6% 氯化钠）在治疗 4 周后导致全肺清除率较基线显着持续改善（p = 0.014），尽管在初始剂量后没有延长的单剂量效应。与安慰剂（0.12% 氯化钠）治疗（p = 0.016）相比，这种持续变化（先前给药后 12 小时）显着更大。HS 对肺活量测定的改善未达到统计学显着性，但与 MCC 变化相关。结论 观察到的 MCC 与 HS 的持续改善表明，这种治疗可能会产生健康益处，即使是对 CF 影响相对较轻的儿童。由于缺乏有意义的、