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Lumacaftor-Ivacaftor Aids Cystic Fibrosis in Real-World Setting

FRIDAY, Oct. 11, 2019 -- After one year of therapy with lumacaftor-ivacaftor in a real-world setting, adolescents and adults with cystic fibrosis and Phe508del homozygous mutation experienced an improvement in lung function, increase in body mass index, and reduced need for IV antibiotics, according to a study published online Oct. 11 in the American Journal of Respiratory and Critical Care Medicine.

Pierre-Régis Burgel, M.D., Ph.D., from Cochin Hospital in Paris, and colleagues examined the safety and effectiveness of lumacaftor-ivacaftor in adolescents and adults in a real-world setting. Data were included from 845 patients with cystic fibrosis and Phe508del homozygous mutation (292 adolescents and 553 adults) who initiated lumacaftor-ivacaftor.

The researchers found that 18.2 percent of patients discontinued treatment, often due to respiratory or nonrespiratory adverse events (48.1 and 27.9 percent, respectively). Adult age group, percent predicted forced expiratory volume in one second (ppFEV1) <40 percent, and numbers of intravenous antibiotic courses during the year before lumacaftor-ivacaftor initiation were associated with increased rates of discontinuation in a multivariable analysis. An absolute increase in ppFEV1 (+3.67 percent), an increase in body mass index (BMI; +0.73 kg/m²), and a 35 percent decrease in intravenous antibiotic courses were seen with continuous exposure to lumacaftor-ivacaftor. A significant decrease in ppFEV1 was seen for patients who discontinued treatment, who also did not improve in BMI or reduce intravenous antibiotic courses.

"Our findings provide a useful complement to randomized clinical trials in which participants were selected based on very strict criteria," Burgel said in a statement.

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Posted: October 2019

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