| Literature DB >> 32513528 |
Michelle Shaw1, Umer Khan2, John P Clancy3, Scott H Donaldson4, Scott D Sagel5, Steven M Rowe6, Felix Ratjen7.
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.Entities:
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Year: 2020 PMID： 32513528 PMCID： PMC9125683 DOI： 10.1016/j.jcf.2020.05.010
Source DB: PubMed Journal: J Cyst Fibros ISSN： 1569-1993 Impact factor: 5.527