Milla Carlos E
Center for Excellence in Pulmonary Biology, Department of Pediatrics, Stanford University, Palo Alto, California, USA.
Curr Opin Pediatr. 2025 Jun 1;37(3):266-271. doi: 10.1097/MOP.0000000000001458. Epub 2025 Mar 27.
The field of cystic fibrosis is experiencing dramatic changes, as the translation of a massive body of scientific knowledge accumulated from the day of the cloning of the CFTR gene has led to the identification of effective therapies to correct the basic defect. This has also allowed care providers and people with cystic fibrosis in low-income and middle-income countries (LMICs) to become more knowledgeable and proficient in cystic fibrosis cares.
This review focuses on two main aspects highly relevant to understand the current status of cystic fibrosis in LMICs: The recognition of the universal occurrence of cystic fibrosis, as well as the varying incidence in different regions of the world, and the collaborative international efforts for dissemination of best care practices as an attempt to close gaps in care.
As the field continues to change rapidly, multiple international efforts are attempting to close gaps and disparities clearly apparent between affluent countries and LMICs. However, these efforts are seriously hampered by limited access to effective therapies and most dramatically to CFTR modulator drugs.
囊性纤维化领域正在经历巨大变革,自CFTR基因克隆之日起积累的大量科学知识转化应用,已促成了纠正基本缺陷的有效疗法的发现。这也使低收入和中等收入国家(LMICs)的医护人员及囊性纤维化患者对该病的护理更有见识且更加熟练。
本综述聚焦于与理解LMICs中囊性纤维化现状高度相关的两个主要方面:认识到囊性纤维化在全球普遍存在,以及在世界不同地区发病率各异,以及国际间为传播最佳护理实践以缩小护理差距而开展的合作努力。
随着该领域持续快速变化,多项国际努力正试图缩小富裕国家与LMICs之间明显存在的差距和差异。然而,这些努力因难以获得有效疗法尤其是CFTR调节剂药物而受到严重阻碍。