{"title":"鲁马卡福/依伐卡福治疗儿童严重囊性纤维化的疗效","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":"1 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"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\":\"1 1\",\"pages\":\"\"},\"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}","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}
Efficacy of lumacaftor/ivacaftor in a child with severe cystic fibrosis
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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