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Ivacaftor safe for pediatric patients with CF, gating mutation
Pediatric patients aged between 2 years and 5 years with cystic fibrosis and a gating mutation who received ivacaftor seemed to tolerate the treatment as well as older children, according to recent research.
“This study confirmed a safe and tolerable dose of an acceptable formulation of ivacaftor in children aged 2 to 5 years with a CFTR gating mutation, although liver function seems to require closer monitoring in this age range than in adults, particularly among those with a history of elevated [liver function tests (LFTs)],” Jane C. Davies, MD, of the National Heart and Lung Institute at the Imperial College London, and colleagues wrote.
As part of the KIWI study, researchers first tested the short-term safety of ivacaftor in 9 patients during a period of four days. Every 12 hours, children less than 14 kg received 50 mg of ivacaftor and children 14 kg or more received 75 mg of ivacaftor, according to the abstract.
The second part of the study evaluated ivacaftor treatment up to 24 weeks. Thirty-four children participated in the second part, including eight patients from the first part of the study.
Davies and colleagues observed similar results in pediatric patients as seen in adult patients (median Cmin = 536 ng/mL, 50 mg; 580 ng/mL, 75 mg) when receiving ivacaftor, according to the abstract. The median area under the curve values were 9,840 ng × h/mL for the 50 mg dose and 10,200 ng × h/mL. The researchers reported 19 of 34 patients (56%) in the second part of the study developed coughing symptoms, and 10 of 34 patients (29%) reported vomiting.
“Six (18%) of 34 patients had seven serious adverse events; a raised concentration of transaminases was the only serious adverse event regarded as related to ivacaftor and the only adverse event that resulted in study treatment discontinuation,” Davies and colleagues wrote.
There were 5 patients (15%) with LFT results significantly higher than normal, which caused an interruption in administering ivacaftor in 4 patients and discontinuation in 1 patient. – by Jeff Craven
Disclosure: Davies reports an advisory board role with Novartis, Pharmaxis, Proteostasis, Pulmocide, and Vertex Pharmaceuticals Incorporated. Please see the full study for a complete list of all other researchers’ relevant financial disclosures.