Ivacaftor for the p.Ser549Arg (S549R) gating mutation - The Israeli experience.

Adi Dagan,Malena Cohen-Cymberknoh,Michal Shteinberg,Hagit Levine,Daphna Vilozni,Yael Bezalel,Bat-El Bar Aluma,Ifat Sarouk,Moshe Ashkenazi,Moran Lavie,Reuven Tsabari,Hannah Blau,Eitan Kerem,Lea Bentur,Ori Efrati,Galit Livnat

Respiratory Medicine（2017）

Cited 18|Views27

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Abstract

•Information on the clinical efficacy of ivacaftor in patients carrying the p.Ser549Arg (S549R) CFTR mutation was sought.•Sweat chloride decreased in all patients, and reached a normal range in 75%.•Lung functions improved in all patients treated: FEV1 improved by 22 ± 14%, FVC by 15 ± 16%; and FEF25-75% by 44 ± 34%.•BMI significantly increased by 10% and there was a significant change in patients’ glucose metabolism.•A reduction in the number of days per year treated with intravenous antibiotics was noted.

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Key words

Cystic fibrosis,Gating mutation,Ivacaftor,p.Ser549Arg (S549R)

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