Abstract
We reviewed the impact of ivacaftor on Scottish paediatric cystic fibrosis (CF)
patients ≥ 6 years of age after 12 months of treatment. Statistically significant
improvements in FEV1 and BMI 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
patients ≥ 6 years of age after 12 months of treatment. Statistically significant
improvements in FEV1 and BMI 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
Original language | English |
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Number of pages | 8 |
Journal | Archives of Disease in Childhood |
Early online date | 10 Jun 2016 |
DOIs | |
Publication status | E-pub ahead of print - 10 Jun 2016 |
Keywords
- cystic fibrosis
- ivacaftor
- G551D
- p.Gly551Asp