Current state of CFTR modulators for treatment of Cystic Fibrosis Katherine A. Despotes and Scott H. Donaldson
Current state of CFTR modulators for treatment of Cystic Fibrosis Katherine A. Despotes and Scott H. Donaldson
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DOI：
10.1016/j.coph.2022.102239
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发表时间：
2022-05-21
影响因子：
4
通讯作者：
Donaldson, Scott H.
中科院分区：
文献类型：
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作者：
Despotes, Katherine A.;Donaldson, Scott H.
囊性纤维化跨膜传导调节蛋白的小分子调节剂正在改变囊性纤维化患者的护理。高效调节剂现已被批准用于近90%的成年CF人群。他们显着改善肺功能，呼吸道症状，并减少肺部恶化。最近的努力正在扩大这些疗法的可用性，以越来越多的儿科患者。调节剂对肺外CF表现的影响各不相同，尽管已经证明营养有了深刻的改善。观察性研究和现实世界的研究表明，治疗的好处是持续随着时间的推移，最大的影响可能会获得早期使用。对于那些不合格突变的患者，需要开发替代方法来恢复囊性纤维化跨膜传导调节因子（CFTR）功能。
Small molecular modulators of the cystic fibrosis transmembrane conductance regulator protein are transforming the care of people with cystic fibrosis. Highly effective modulators are now approved for nearly 90% of the adult CF population. They dramatically improve lung function, respiratory symptoms, and reduce pulmonary exacerbations. Recent efforts are expanding the availability of these therapies to a growing number of pediatric patients. The impact of modulators on extrapulmonary CF manifestations varies, although profound improvements in nutrition have been demonstrated. Observational studies and real-world research suggest that treatment benefits are sustained over time, and that maximal impact may be obtained with early use. The development of alternative approaches to restoring cystic fibrosis transmembrane conductance regulator (CFTR) function is needed for those with ineligible mutations.