Combination therapy with elexacaftor/tezacaftor/ivacaftor resulted in clinically significant improvements in lung function in patients with cystic fibrosis, according to the results of a recent study published in the European Respiratory Journal.

Researchers retrospectively reviewed pulmonary function parameters from a cohort of patients with cystic fibrosis taking combination therapy consisting of elexacaftor/tezacaftor/ivacaftor for a period of 1 year. Outcomes of interest included changes in forced expiratory volume in 1 second percent predicted (FEV1) from baseline, patient weight and body mass index (BMI), and rate of pulmonary exacerbations.

Among the 22 patients with advanced lung disease, the average age was 37.2 years and mean baseline FEV1 percent predicted was 27.7%. One month after treatment, spirometry measurements showed that FEV1 percent predicted increased by 5.5% from baseline (P <.001). At the 12 to 15 month follow-up, the FEV1 percent predicted increased by 7.6% from baseline (P <.001). At 1 month, mean weight increased by 1.74 kg and mean BMI increased 0.7 and at 12 months, weight increased to 5.36 kg and BMI increased by 2.0.

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In the 12 months before treatment, patients experienced an average of 3.76 pulmonary exacerbations, but 1.38 exacerbations during the 12 months on elexacaftor/tezacaftor/ivacaftor (P <.001). Mean respiratory domain scores increased 24.1 points (P =.006) over 1 month of treatment and 45.6 points (P =.003) over 6 months of treatment.

In terms of tolerability, 6 patients reported symptoms that may have been associated with elexacaftor/tezacaftor/ivacaftor, including rash, ankle edema, worsened cough, and abdominal pain. None of the patients discontinued treatment.

“The well-tolerated [cystic fibrosis transmembrane regulator] modulator combination of [elexacaftor/tezacaftor/ivacaftor] was associated with clinically significant improvements in cystic fibrosis patients with advanced lung disease,” the study authors wrote. “Our experience indicates this impactful treatment will ultimately delay need for lung transplantation.”

Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


O’Shea KM, O’Carroll OM, Carroll C, et al. Efficacy of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease. Eur Respir J. 2021;57(2):2003079. doi:10.1183/13993003.03079-2020