Yale Adult Cystic Fibrosis Program, Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Internal Medicine, Yale University School of Medicine.
Division of Respiratory Medicine, Translational Medicine, University of Toronto Hospital for Sick Children, 555 University Avenue, Toronto Ontario M5G 1X8, Canada.
Clin Chest Med. 2022 Dec;43(4):631-646. doi: 10.1016/j.ccm.2022.06.006.
As we characterize the clinical benefits of highly effective modulator therapy (HEMT) in the cystic fibrosis (CF) population, our paradigm for treating and monitoring disease continues to evolve. More sensitive approaches are necessary to detect early disease and clinical progression. This article reviews evolving strategies to assess disease control and progression in the HEMT era. This article also explores developments in pulmonary function monitoring, advanced respiratory imaging, tools for the collection of patient-reported outcomes, and their application to profile individual responses, guide therapeutic decisions, and improve the quality of life of people with CF.
随着我们描述高效调节剂治疗 (HEMT) 在囊性纤维化 (CF) 人群中的临床益处,我们治疗和监测疾病的模式也在不断发展。需要更敏感的方法来早期发现疾病和临床进展。本文综述了在 HEMT 时代评估疾病控制和进展的不断发展的策略。本文还探讨了肺功能监测、先进呼吸成像、患者报告结果的收集工具的发展,以及它们在描绘个体反应、指导治疗决策和提高 CF 患者生活质量方面的应用。