Ratjen Felix
Department of Pediatrics, Division of Respiratory Medicine, Ontario, Canada.
Translational Medicine Program, Research Institute, Hospital for Sick Children, Ontario, Canada.
Pediatr Pulmonol. 2025 Mar;60 Suppl 1:S75-S76. doi: 10.1002/ppul.27337. Epub 2024 Oct 17.
The severity of lung disease as well as other disease manifestations have dramatically improved in those patients with cystic fibrosis (CF) that both have mutations responsive to small molecule- based therapies with CF transmembrane regulator (CFTR) modulators and do have access to these drugs. Unfortunately, these medications are not available to many patients with CF across the globe with access largely limited to high income countries. For those eligible to CFTR modulators new questions have arisen regarding the ongoing need for other medications addressing CF lung disease as well current care models with tight monitoring. This article aims to summarize how CF care may change in the future making a plea to expand the availability of highly effective medications to every child with CF that could benefit from treatment.
对于那些患有囊性纤维化(CF)且具有对基于小分子疗法的CF跨膜调节因子(CFTR)调节剂有反应的突变并且能够获得这些药物的患者,肺部疾病以及其他疾病表现的严重程度已得到显著改善。不幸的是,全球许多CF患者无法获得这些药物,药物获取在很大程度上仅限于高收入国家。对于那些有资格使用CFTR调节剂的患者,出现了关于持续需要其他治疗CF肺部疾病的药物以及当前严格监测的护理模式的新问题。本文旨在总结CF护理在未来可能如何变化,呼吁扩大高效药物的可及性,使每个能从治疗中受益的CF患儿都能获得药物。