在婴幼儿囊性纤维化患者中吸入高渗盐水：短期耐受性、依从性和安全性。
Inhaled hypertonic saline in infants and toddlers with cystic fibrosis: short-term tolerability, adherence, and safety.
机构信息
Department of Pediatrics, University of Washington School of Medicine, Seattle Children's Hospital, 4800 Sandpoint Way NE, Seattle, WA 98105, USA.
出版信息
Pediatr Pulmonol. 2011 Jul;46(7):666-71. doi: 10.1002/ppul.21425. Epub 2011 Mar 1.
BACKGROUND
Inhaled hypertonic saline (HS) is an attractive agent for chronic maintenance therapy in infants and toddlers with cystic fibrosis (CF) because it improves defective mucociliary clearance. Prior to undertaking a clinical trial of HS efficacy in young children with CF, tolerability, adherence, and safety must be established.
METHODS
Three-center, open label evaluation of the short-term tolerability, adherence, and safety of 7% HS administered twice daily for 14 days in children with CF 12-30 months of age. The primary objective was to evaluate the proportion of participants unable to tolerate single and repeated doses of 7% HS according to protocol-defined criteria. Participants inhaled a test dose of HS at the enrollment visit; test dose intolerance was defined as fulfillment of at least one of 4 criteria. Participants who tolerated the test dose inhaled 7% HS twice daily for 14±2 days.
RESULTS
Twenty children were enrolled. One was withdrawn due to maternal concern over fussiness with application of the facemask for the test dose. Of the 19 participants administered the test dose, 1 was withdrawn due to test dose intolerance (5%, 95% confidence interval 0, 26%). Eighteen participants completed the study; 1 was intolerant (95% CI 0, 27%) at the final visit due to new wheezes on exam in association with an upper respiratory infection and otitis media. Home symptom diaries demonstrated cough as the main symptom in the hour following inhalation, which decreased in frequency over the study period. Adherence as assessed by daily home diary and returned study drug ampoules was high. Participants reported receiving both treatments on a median of 100% of days; a median of 25 ampoules were used during a median of 13 days.
CONCLUSIONS
7% HS appears well tolerated for up to 14 days in infants and toddlers with CF, with high adherence. These results provide encouraging short-term tolerability and adherence data for future trials assessing the safety and efficacy of 7% HS in young children with CF.
背景
吸入高渗盐水（HS）是囊性纤维化（CF）婴幼儿慢性维持治疗的一种有吸引力的药物，因为它可以改善缺陷的黏液纤毛清除功能。在进行 HS 对 CF 幼儿疗效的临床试验之前，必须确定其耐受性、依从性和安全性。
方法
在年龄为 12-30 个月的 CF 儿童中，进行为期 14 天、每天两次给予 7% HS 的短期耐受性、依从性和安全性的三中心、开放性评估。主要目的是根据方案定义的标准评估无法耐受单次和重复剂量 7% HS 的参与者比例。参与者在入组就诊时吸入 HS 测试剂量；测试剂量不耐受定义为满足至少 4 项标准中的 1 项。耐受测试剂量的参与者每天两次吸入 7% HS 共 14±2 天。
结果
共有 20 名儿童入组。由于母亲担心面罩应用时孩子烦躁，1 名儿童退出研究。在接受测试剂量的 19 名参与者中，因测试剂量不耐受（5%，95%置信区间 0，26%）而退出 1 名。18 名参与者完成了研究；1 名在最后一次就诊时不耐受（95%CI 0，27%），原因是上呼吸道感染和中耳炎引起的检查时新出现的喘息。家庭症状日记显示，吸入后 1 小时内主要症状为咳嗽，研究期间咳嗽频率逐渐降低。通过每日家庭日记和回收的研究药物安瓿评估，依从性很高。参与者报告中位数为 100%的天数接受了两种治疗，中位数为 13 天使用了中位数为 25 支安瓿。
结论
7% HS 在 CF 婴儿和幼儿中最长可耐受 14 天，依从性高。这些结果为未来评估 7% HS 在 CF 幼儿中的安全性和疗效的试验提供了令人鼓舞的短期耐受性和依从性数据。
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