Rowe Steven M, Clancy John P
Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Curr Opin Pediatr. 2006 Dec;18(6):604-13. doi: 10.1097/MOP.0b013e3280109b90.
Over the past four decades, outcomes for patients with cystic fibrosis have improved dramatically. Major contributors to this improvement are a better understanding of disease pathogenesis and the systematic conduct of clinical trials evaluating new therapies designed to address these defects. This review describes recent developments in cystic fibrosis pulmonary therapies intended to treat various facets of the disease, including several treatments currently in development.
The mainstays of therapy for cystic fibrosis, such as nutritional support and mechanical mucus clearance, are now supplemented with aggressive antibiotic regimens intended to suppress or eradicate bacterial colonization, anti-inflammatory agents, and new approaches that improve mucociliary clearance. Therapies in development address the underlying ion transport defect found in cystic fibrosis airways and also include small-molecule agents that restore function to the mutant cystic fibrosis transmembrane conductance regulator.
Recent advances in therapies for cystic fibrosis offer the promise of improved outcomes and longer lives for patients with cystic fibrosis.
在过去四十年中，囊性纤维化患者的治疗结果有了显著改善。这一改善的主要因素是对疾病发病机制有了更深入的了解，以及系统开展了评估旨在解决这些缺陷的新疗法的临床试验。本综述描述了囊性纤维化肺部治疗的最新进展，这些治疗旨在治疗该疾病的各个方面，包括目前正在研发的几种治疗方法。
囊性纤维化的主要治疗方法，如营养支持和机械性排痰，现在辅以旨在抑制或根除细菌定植的积极抗生素方案、抗炎药物以及改善黏液纤毛清除功能的新方法。正在研发的治疗方法针对囊性纤维化气道中存在的潜在离子转运缺陷，还包括能恢复突变型囊性纤维化跨膜传导调节因子功能的小分子药物。
囊性纤维化治疗的最新进展为囊性纤维化患者带来了改善治疗结果和延长生命的希望。
