囊性纤维化患者能否最终借助鲁马卡托/依伐卡托顺畅呼吸？
Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
作者信息
Schneider E K, Reyes-Ortega F, Li J, Velkov T
机构信息
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.
Monash Biomedicine Discovery Institute, Department of Microbiology, Monash University, Clayton, Victoria, Australia.
出版信息
Clin Pharmacol Ther. 2017 Jan;101(1):130-141. doi: 10.1002/cpt.548. Epub 2016 Nov 23.
Abstract
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the "magic-bullet" cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
摘要
囊性纤维化（CF）是一种由囊性纤维化跨膜传导调节因子（CFTR）活性缺陷或不足引起的危及生命的疾病。美国食品药品监督管理局（FDA）最近批准的鲁马卡托与依伐卡托联合用药（Orkambi）针对的是携带F508del-CFTR突变的患者。问题依然存在：这种突破性的联合疗法对绝大多数囊性纤维化患者来说是否是“万灵药”？这篇综述涵盖了鲁马卡托/依伐卡托的当代临床和科学知识基础，并突出了近期相互矛盾的文献报告中出现的新问题。
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本文引用的文献
1
Development of HPLC and LC-MS/MS methods for the analysis of ivacaftor, its major metabolites and lumacaftor in plasma and sputum of cystic fibrosis patients treated with ORKAMBI or KALYDECO.
J Chromatogr B Analyt Technol Biomed Life Sci. 2016 Dec 1;1038:57-62. doi: 10.1016/j.jchromb.2016.10.026. Epub 2016 Oct 24.
2
An "Unlikely" Pair: The Antimicrobial Synergy of Polymyxin B in Combination with the Cystic Fibrosis Transmembrane Conductance Regulator Drugs KALYDECO and ORKAMBI.
ACS Infect Dis. 2016 Jul 8;2(7):478-88. doi: 10.1021/acsinfecdis.6b00035. Epub 2016 May 17.
3
Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation.
Expert Rev Precis Med Drug Dev. 2016;1(3):235-243. doi: 10.1080/23808993.2016.1175299. Epub 2016 Apr 22.
4
Social/economic costs and health-related quality of life in patients with cystic fibrosis in Europe.
Eur J Health Econ. 2016 Apr;17 Suppl 1:7-18. doi: 10.1007/s10198-016-0781-6. Epub 2016 Apr 8.
5
CFTR modulators and pregnancy: Our work has only just begun.
J Cyst Fibros. 2016 Jan;15(1):6-7. doi: 10.1016/j.jcf.2015.12.019. Epub 2016 Jan 7.
6
A successful uncomplicated CF pregnancy while remaining on Ivacaftor.
J Cyst Fibros. 2016 Jan;15(1):133-4. doi: 10.1016/j.jcf.2015.11.013. Epub 2015 Dec 14.
7
Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction.
Expert Rev Respir Med. 2016;10(1):5-17. doi: 10.1586/17476348.2016.1122527. Epub 2015 Dec 9.
8
Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell.
Eur J Pediatr. 2016 Jan;175(1):1-8. doi: 10.1007/s00431-015-2664-8. Epub 2015 Nov 14.
9
Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor).
Br J Pharmacol. 2016 Feb;173(3):459-70. doi: 10.1111/bph.13365. Epub 2016 Jan 13.
10
Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?
Thorax. 2015 Jul;70(7):615-6. doi: 10.1136/thoraxjnl-2015-207369.
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