Results are out from a two part study involving children ages 2-5 with at least one gating mutation trying Ivacaftor (a medication used in Cystic Fibrosis treatment)
From the Lancet Respiratory Journal, 20 January 2016
Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in patients aged 6 years or older with a CFTR gating mutation. We aimed to assess the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in children aged 2–5 years.
In the two-part KIWI study, we enrolled children aged 2–5 years weighing 8 kg or more with a confirmed diagnosis of cystic fibrosis and a CFTR gating mutation on at least one allele from 15 hospitals in the USA, UK, and Canada. Read more
Ivacaftor at doses of 50 mg and 75 mg seems to be safe in children aged 2–5 years with cystic fibrosis with a gating mutation followed up for 24 weeks, although the frequency of elevated LFTs suggests that monitoring should be frequent in young children, particularly those with a history of elevated LFTs. Results of an ongoing extension study assessing durability of these effects and longer-term safety are warranted.