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囊性纤维化领域新时代的肺移植

Lung Transplantation in a New Era in the Field of Cystic Fibrosis.

作者信息

Huang Wei, Smith Alexander T, Korotun Maksim, Iacono Aldo, Wang Janice

机构信息

Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY 11549, USA.

Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY 11549, USA.

出版信息

Life (Basel). 2023 Jul 21;13(7):1600. doi: 10.3390/life13071600.

Abstract

Lung transplantation for people with cystic fibrosis (PwCF) is a critical therapeutic option, in a disease without a cure to this day, and its overall success in this population is evident. The medical advancements in knowledge, treatment, and clinical care in the field of cystic fibrosis (CF) rapidly expanded and improved over the last several decades, starting from early pathology reports of CF organ involvement in 1938, to the identification of the CF gene in 1989. Lung transplantation for CF has been performed since 1983, and CF now accounts for about 17% of pre-transplantation diagnoses in lung transplantation recipients. Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been the latest new therapeutic modality addressing the underlying CF protein defect with the first modulator, ivacaftor, approved in 2012. Fast forward to today, and we now have a growing CF population. More than half of PwCF are now adults, and younger patients face a better life expectancy than they ever did before. Unfortunately, CFTR modulator therapy is not effective in all patients, and efficacy varies among patients; it is not a cure, and CF remains a progressive disease that leads predominantly to respiratory failure. Lung transplantation remains a lifesaving treatment for this disease. Here, we reviewed the current knowledge of lung transplantation in PwCF, the challenges associated with its implementation, and the ongoing changes to the field as we enter a new era in the care of PwCF. Improved life expectancy in PwCF will surely influence the role of transplantation in patient care and may even lead to a change in the demographics of which people benefit most from transplantation.

摘要

对于患有囊性纤维化(CF)的患者(PwCF)而言,肺移植是一种关键的治疗选择,因为至今尚无治愈该疾病的方法,且肺移植在这一人群中的整体成效显著。在过去几十年里,囊性纤维化(CF)领域在知识、治疗和临床护理方面的医学进步迅速发展并不断改善,从1938年CF器官受累的早期病理报告,到1989年CF基因的鉴定。自1983年以来一直开展针对CF的肺移植,如今CF在肺移植受者移植前诊断中约占17%。囊性纤维化跨膜传导调节因子(CFTR)调节剂是解决CF潜在蛋白质缺陷的最新治疗方式,首款调节剂依伐卡托于2012年获批。快进到如今,CF患者群体不断壮大。现在超过一半的PwCF是成年人,年轻患者的预期寿命比以往任何时候都要长。不幸的是,CFTR调节剂疗法并非对所有患者都有效,疗效在患者之间存在差异;它并非治愈方法,CF仍是一种主要导致呼吸衰竭的进行性疾病。肺移植仍然是这种疾病的一种挽救生命的治疗方法。在此,我们回顾了PwCF肺移植的现有知识、实施过程中面临的挑战,以及随着我们进入PwCF护理的新时代该领域正在发生的变化。PwCF预期寿命的提高肯定会影响移植在患者护理中的作用,甚至可能导致从移植中获益最大的人群的人口统计学特征发生变化。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d40c/10381966/640d51711a44/life-13-01600-g001.jpg

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