迈向建立囊性纤维化新的临床终点：肺清除指数和心肺运动试验的作用。
Toward the Establishment of New Clinical Endpoints for Cystic Fibrosis: The Role of Lung Clearance Index and Cardiopulmonary Exercise Testing.
作者信息
Hatziagorou Elpis, Kampouras Asterios, Avramidou Vasiliki, Toulia Ilektra, Chrysochoou Elisavet-Anna, Galogavrou Maria, Kirvassilis Fotios, Tsanakas John
机构信息
Pediatric Pulmonology and Cystic Fibrosis Unit, Hippokration Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
出版信息
Front Pediatr. 2021 Feb 25;9:635719. doi: 10.3389/fped.2021.635719. eCollection 2021.
Abstract
As Cystic Fibrosis (CF) treatment advances, research evidence has highlighted the value and applicability of Lung Clearance Index and Cardiopulmonary Exercise Testing as endpoints for clinical trials. In the context of these new endpoints for CF trials, we have explored the use of these two test outcomes for routine CF care. In this review we have presented the use of these methods in assessing disease severity, disease progression, and the efficacy of new interventions with considerations for future research.
摘要
随着囊性纤维化（CF）治疗的进展，研究证据凸显了肺清除指数和心肺运动试验作为临床试验终点的价值和适用性。在CF试验的这些新终点的背景下，我们探讨了将这两种测试结果用于CF常规护理的情况。在本综述中，我们介绍了这些方法在评估疾病严重程度、疾病进展以及新干预措施疗效方面的应用，并对未来研究进行了思考。
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A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.一项 3 期、双盲、平行分组研究，旨在评估 tezacaftor 联合 ivacaftor 在 6 至 11 岁囊性纤维化纯合子 F508del 或杂合子 F508del-CFTR 突变和残余功能突变的参与者中的疗效和安全性。
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