Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del<i>CFTR</i>
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- 0.276 · how far apart the two teachers sit on this one work
- Validation status
score_only:v0-immature-baseline· verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it
Abstract
BACKGROUND: Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis. METHODS: through week 24 (calculated as a percentage) was a key secondary end point. RESULTS: in favor of tezacaftor-ivacaftor over placebo were 4.0 percentage points and 6.8%, respectively (P<0.001 for both comparisons). The rate of pulmonary exacerbation was 35% lower in the tezacaftor-ivacaftor group than in the placebo group (P=0.005). The incidence of adverse events was similar in the two groups. Most adverse events were of mild severity (in 41.8% of patients overall) or moderate severity (in 40.9% overall), and serious adverse events were less frequent with tezacaftor-ivacaftor (12.4%) than with placebo (18.2%). A total of 2.9% of patients discontinued the assigned regimen owing to adverse events. Fewer patients in the tezacaftor-ivacaftor group than in the placebo group had respiratory adverse events, none of which led to discontinuation. CONCLUSIONS: The combination of tezacaftor and ivacaftor was efficacious and safe in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation. (Funded by Vertex Pharmaceuticals; EVOLVE ClinicalTrials.gov number, NCT02347657 .).
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The record
- Venue
- New England Journal of Medicine
- Topic
- Cystic Fibrosis Research Advances
- Field
- Medicine
- Canadian institutions
- SickKids FoundationHospital for Sick ChildrenUniversity of Toronto
- Funders
- National Center for Advancing Translational SciencesNational Center for Research ResourcesNational Institute of Diabetes and Digestive and Kidney Diseases
- Keywords
- IvacaftorMedicineCystic fibrosisCystic fibrosis transmembrane conductance regulatorPlaceboRandomizationInternal medicineClinical endpointGastroenterologyRandomized controlled trialUrologySurgeryPathology
- Has abstract in OpenAlex
- yes