Patients with cystic fibrosis (CF) who were treated for 1 year with a combination of lumacaftor and ivacaftor saw improvements in glucose tolerance abnormalities, according to the results of a study published in the Journal of Cystic Fibrosis.

Oral glucose tolerance tests were used to evaluate baseline abnormalities in glucose tolerance in patients with CF with either glucose intolerance or newly diagnosed diabetes. Patients were then treated with a combination of lumacaftor and ivacaftor for a period of 1 year and glucose tolerance was retested and compared with baseline.

Of the 40 patients with CF included in the study, 78% had glucose intolerance and 22% had newly diagnosed diabetes. After 1 year of treatment, 50% patients had glucose tolerance classified as normal, 40% had glucose intolerance, and 10% had diabetes. Glucose tolerance improved in 57.5% of patients, with a significant decrease in both 1 and 2-hour oral glucose tolerance test glycemia.

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The main study limitations included a small sample size as well as the nonrandomized design.

“[A] positive lumacaftor-ivacaftor treatment impact was demonstrated in CF patients with early [abnormalities in glucose tolerance],” the researchers wrote.  “Larger longitudinal studies are needed to better define the role of CF [transmembrane conductance regulator] modulators in CF.”

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Disclosure: This study was supported by Vertex Pharmaceuticals. Please see the original reference for a full list of authors’ disclosures.


Misgault B, Chatron E, Reynaud Q, et al. Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients [published online March 19, 2020]. J Cyst Fibros. doi:10.1016/j.jcf.2020.03.002