Henke Markus O, Ratjen Felix
Department of Pulmonary Medicine, Philipps-University Marburg, 35043 Marburg, Germany.
Paediatr Respir Rev. 2007 Mar;8(1):24-9. doi: 10.1016/j.prrv.2007.02.009. Epub 2007 Mar 21.
Mucus accumulation in the lower airways is a key feature of cystic fibrosis (CF) lung disease. The major component of mucus in CF is not mucin derived from mucus producing cells but rather pus that includes viscous material such as polymerized DNA derived from degraded neutrophils. This has important implications for mucolytic therapy aiming to improve mucus clearance from the airways, since degradation of mucin may not be a suitable treatment strategy. In addition, thinning of secretions may not always be beneficial, since it may negatively affect certain aspects of mucus transport such as cough clearance. While inhaled N-acetylcysteine has been used as a mucolytic drug in CF for decades, there is little evidence that it has any beneficial effect. Dornase alfa has been shown to reduce pulmonary exacerbations and improve lung function and is currently the only mucolytic agent with proven efficacy in CF. Newer agents targeting other components of CF mucus, such as filamentous actin, are currently in development. Ultimately, drugs that are mucokinetic, which preserve viscoelasticity, rather than mucolytic may prove to be beneficial for CF lung disease in the future.
下呼吸道黏液积聚是囊性纤维化（CF）肺部疾病的一个关键特征。CF患者黏液的主要成分并非来自黏液分泌细胞的黏蛋白，而是脓液，其中包括诸如源自降解中性粒细胞的聚合DNA等黏性物质。这对于旨在改善气道黏液清除的黏液溶解疗法具有重要意义，因为降解黏蛋白可能不是一种合适的治疗策略。此外，分泌物变稀并不总是有益的，因为这可能会对黏液运输的某些方面产生负面影响，如咳嗽清除。虽然吸入式N - 乙酰半胱氨酸在CF治疗中作为黏液溶解药物已使用数十年，但几乎没有证据表明它有任何有益效果。脱氧核糖核酸酶α已被证明可减少肺部病情加重并改善肺功能，目前是CF中唯一已证实有效的黏液溶解剂。目前正在研发针对CF黏液其他成分（如丝状肌动蛋白）的新型药物。最终，具有促黏液运动功能、能保持黏弹性而非具有黏液溶解作用的药物，未来可能被证明对CF肺部疾病有益。
