The use of lumacaftor/ivacaftor to treat acute deterioration in paediatric cystic fibrosis

被引：3
作者：
Hammond, James A. [1 ]
Connett, Gary J. [1 ]
机构：
[1] Southampton Childrens Hosp, Southampton Biomed Res Ctr, Natl Inst Hlth Res, Tremona Rd, Southampton SO16 6YD, Hants, England
来源：
PAEDIATRIC RESPIRATORY REVIEWS | 2018年 / 27卷
关键词：
Cystic fibrosis; Lumacaftor; Ivacaftor; Paediatric; SEVERE LUNG-DISEASE; AGED; 6-11; YEARS; COMBINATION THERAPY; PHE508DEL CFTR; IN-VITRO; F508DEL-CFTR; INITIATION; IVACAFTOR; EFFICACY; MUTATION;
D O I：
10.1016/j.prrv.2018.05.008
中图分类号：
R72 [儿科学];
学科分类号：
100202 ;
摘要：
Lumacaftor/ivacaftor is a precision medicine targeting the defective cystic fibrosis transmembrane regulator (CFTR) protein in cystic fibrosis (CF) patients homozygous for Phe508del genotype. Whilst there is evidence for efficacy in children aged 6-11 years who are stable with good lung function, there are little data about the use of this medication for children with acute deterioration in this age group. We describe the use of this drug to treat a child with an unusually severe exacerbation of CF lung disease and review the potential of lumacaftor/ivacaftor as a rescue therapy in the paediatric CF population. Crown Copyright (C) 2018 Published by Elsevier Ltd. All rights reserved.
引用
收藏
页码：16 / 17
页数：2
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