The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation: a review.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 27288428.
- Also identified by DOI 10.1136/archdischild-2015-310420.
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Abstract
We reviewed the impact of ivacaftor on Scottish paediatric patients with cystic fibrosis ≥6 years of age after 12 months of treatment. Statistically significant improvements in FEV<sub>1</sub> and body mass index and a reduction in sweat chloride, all comparable with previously published data were observed. The findings also suggested reduced use of intravenous antibiotics and oral antibiotics. No significant adverse effects were observed but a possible association with cataract formation could not be excluded. This review suggests that, in the short term at least, ivacaftor is effective and safe in paediatric patients ≥6 years of age with G551D.
Medical subject headings
- Aminophenols
- Aminophenols/therapeutic use
- Child
- Chloride Channel Agonists
- Chloride Channel Agonists/therapeutic use
- Cystic Fibrosis
- Cystic Fibrosis/drug therapy
- Cystic Fibrosis Transmembrane Conductance Regulator
- Cystic Fibrosis Transmembrane Conductance Regulator/genetics
- Humans
- Mutation
- Quinolones
- Quinolones/therapeutic use
- Scotland
- Treatment Outcome