Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
Michelle ShawPROSPECT Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network

Abstract

The PROSPECT study, a post-approval observational study in the U.S., showed no significant changes in lung function as measured by spirometry with clinical initiation of lumacaftor/ivacaftor. A sub-study within the PROSPECT study assessed the lung clearance index (LCI), as measured by multiple breath washout (MBW), a measure of lung function demonstrated to be sensitive among people with normal spirometry. Participants performed MBW prior to clinically initiating lumacaftor/ivacaftor therapy and for one year of follow-up. Similar to the whole PROSPECT study, this sub-study cohort (N = 49) had no significant absolute or relative changes in FEV1% predicted at any time point. LCI, however, decreased (improved) by 0.81 units or 5.3% (95% CI -9.7, -0.9%) at 1 month, 0.77 units or 5.9% at 3 months, 0.67 units or 5.9% at 6 months, and 0.55 units or 4.3% at 12 months. These results demonstrate the utility of the LCI in assessing treatment effects of relatively modest size in a heterogenous study population.

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Citations

Sep 15, 2020·Current Opinion in Pulmonary Medicine·Claire NissenbaumJane C Davies
Nov 12, 2020·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·C ShortJ C Davies
Feb 24, 2021·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Dave P NicholsSteven M Rowe
Jun 5, 2021·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Scott H DonaldsonWilliam D Bennett
Jun 30, 2021·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Philippe ReixUNKNOWN French Cystic Fibrosis Reference Network study group
Aug 8, 2021·Italian Journal of Pediatrics·Vito TerlizziDiletta Innocenti
Aug 19, 2021·Pediatric Pulmonology·Danielle M Goetz, Adrienne P Savant

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