Efficacy of ivacaftor in a child with cystic fibrosis, end-stage lung disease, and cepacia syndrome

Walter A. Castro-Elias; Shipra Singh; Peter W. Hiatt; Christopher M. Oermann; Robert H. Moore

doi:10.1089/ped.2012.0193

Efficacy of ivacaftor in a child with cystic fibrosis, end-stage lung disease, and cepacia syndrome

Walter A. Castro-Elias
, Shipra Singh
, Peter W. Hiatt
, Christopher M. Oermann
, Robert H. Moore

Department of Pediatrics

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Abstract

Ivacaftor is a small-molecule potentiator of the cystic fibrosis (CF) transmembrane-conductance regulator and was recently approved for use in CF patients with at least one copy of the G551D mutation. Two clinical trials have proven its efficacy in CF patients 6 years of age and older with mild-to-moderate lung disease. In this article, we report the efficacy of ivacaftor in a 12-year-old CF patient with very severe lung disease and clinical features of cepacia syndrome.

Original language	English (US)
Pages (from-to)	231-233
Number of pages	3
Journal	Pediatric, Allergy, Immunology, and Pulmonology
Volume	25
Issue number	4
DOIs	https://doi.org/10.1089/ped.2012.0193
State	Published - Dec 1 2012

All Science Journal Classification (ASJC) codes

Pediatrics, Perinatology, and Child Health
Immunology and Allergy
Pulmonary and Respiratory Medicine

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10.1089/ped.2012.0193

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AU - Oermann, Christopher M.

AU - Moore, Robert H.

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Efficacy of ivacaftor in a child with cystic fibrosis, end-stage lung disease, and cepacia syndrome

Abstract

All Science Journal Classification (ASJC) codes

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