{"title":"Efficacy of lumacaftor/ivacaftor in a child with severe cystic fibrosis","authors":"A. Goryainova, S. Semykin, P. Mikhalaki","doi":"10.20953/1817-7646-2022-3-136-140","DOIUrl":null,"url":null,"abstract":"We report a case of successful treatment of severe mixed cystic fibrosis in a 11-year-old child using lumacaftor 100 mg/ivacaftor 125mg, a CFTR modulator. This case shows that lumacaftor/ivacaftor is effective in patients who already developed chronic respiratory failure and have low basal FEV1. Key words: CFTR modulators, lumacaftor/ivacaftor, cystic fibrosis","PeriodicalId":38157,"journal":{"name":"Voprosy Prakticheskoi Pediatrii","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Voprosy Prakticheskoi Pediatrii","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.20953/1817-7646-2022-3-136-140","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0
Abstract
We report a case of successful treatment of severe mixed cystic fibrosis in a 11-year-old child using lumacaftor 100 mg/ivacaftor 125mg, a CFTR modulator. This case shows that lumacaftor/ivacaftor is effective in patients who already developed chronic respiratory failure and have low basal FEV1. Key words: CFTR modulators, lumacaftor/ivacaftor, cystic fibrosis
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