A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis.
A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis.
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雾化阿米洛利治疗囊性纤维化肺部疾病的初步研究。
DOI：
10.1056/nejm199004263221704
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发表时间：
1990
期刊：
影响因子：
--
通讯作者：
Boucher,RC
中科院分区：
文献类型：
--
作者：
Knowles,MR;Church,NL;Waltner,WE;Yankaskas,JR;Gilligan,P;King,M;Edwards,LJ;Helms,RW;Boucher,RC
钠的过度主动吸收是囊性纤维化患者气道上皮的独特异常。这种缺陷与粘液增厚和气道分泌物清除不良有关，并可能导致这些患者的肺部疾病。为了研究抑制钠的过度吸收是否会影响囊性纤维化患者肺部疾病的病程，我们进行了一项双盲交叉试验，比较雾化阿米洛利（每升5 mmol; 3.5 ml，每日4次），一种钠通道阻滞剂，与单独的车辆。最初参加研究的18名成人患者中有14名完成了为期一年的试验（每次治疗25周）。用力肺活量（FVC）的平均（±SEM）损失从单独溶剂治疗期间的每天3.39±1.13 ml减少到阿米洛利治疗期间的每天1.44±0.67 ml（P<0.04）。痰液粘度和弹性的测量指标在单独使用赋形剂治疗期间异常，在使用阿米洛利治疗期间改善。在阿米洛利治疗期间，计算的粘液纤毛和咳嗽清除指数也得到改善。阿米洛利无全身、呼吸道或主观毒性作用，我们从初步研究中得出结论，阿米洛利雾化吸入可以安全地用于囊性纤维化成人患者。FVC下降减慢，痰液粘稠度和弹性改善，提示了有益的临床效果。雾化阿米洛利值得进一步评估在囊性纤维化患者肺部疾病的治疗。（N Engl J Med 1990; 322：1189- 1194.）
Excessive active absorption of sodium is a unique abnormality of the airway epithelium in patients with cystic fibrosis. This defect is associated with thickened mucus and poor clearance of airway secretions and may contribute to the pulmonary disease in these patients. To study whether the inhibition of excessive absorption of sodium might affect the course of lung disease in cystic fibrosis, we performed a double-blind, crossover trial comparing aerosolized amiloride (5 mmol per liter; 3.5 ml four times daily), a sodium-channel blocker, with vehicle alone.Fourteen of the 18 adult patients initially enrolled in the study completed the one-year trial (25 weeks for each treatment). The mean (±SEM) loss of forced vital capacity (FVC) was reduced from 3.39±1.13 ml per day during treatment with vehicle alone to 1.44±0.67 ml per day during treatment with amiloride (P<0.04). A measured index of sputum viscosity and elasticity was abnormal during treatment with vehicle alone and improved during treatment with amiloride. Calculated indexes of mucociliary and cough clearance also improved during amiloride treatment. No systemic, respiratory, or subjective toxic effects of amiloride were noted.We conclude from this preliminary study that aerosolized amiloride can be safely administered to adults with cystic fibrosis. The slowing of the loss of FVC and the improvement in sputum viscosity and elasticity suggest a beneficial clinical effect. Aerosolized amiloride deserves further evaluation in the treatment of lung disease in patients with cystic fibrosis. (N Engl J Med 1990; 322: 1189–94.)