|Title:||The efficacy of Elexacaftor/Tezacaftor/Ivacaftor in patients with cystic fibrosis and advanced lung disease|
|Author(s):||O.M. O'Carroll K.M.O'Shea C.Carroll B. Grogan A.Connolly L. O'Shaughnessy T. Nicholson C.G. Gallagher E.F. McKone|
|Institution:||National Referral Centre for Adult Cystic Fibrosis, St. Vincent’s University Hospital, Dublin 4, Ireland.|
|Poster:||Click to view poster|
|Abstract:||Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has delivered transformative improvements to the lives of people with mild to moderate cystic fibrosis with at least one Phe508del mutation (1, 2). The effects of this drug combination are not yet well understood in patients with severe disease.|
We conducted a single-centre observational study with a cohort of patients who received (ELX/TEZ/IVA) as part of a managed access programme if they had either severe lung disease (ppFEV1<40%) or were awaiting lung transplantation. Multiple outcomes were measured and compared with baseline data. Paired student T-test was used and a two-sided p-value of <0.05 was considered to be significant.
14 patients were included in this analysis. After treatment with ELX/TEX/IVA was initiated, ppFEV1 improved (27.37.3% vs 36.3 16.5%, p<0.0001, n=14). BMI also improved (20.73.6 vs 22.13.4 kg/m2, p<0.0001) as did sweat chloride concentration (105.715.9 vs 5019.8 mmol/l, p<0.0001). Infective exacerbations requiring hospitalisation reduced in frequency (0.28 0.17 exacerbations per month vs 0.04 0.07 exacerbations per month, p<0.001) (Figure 1).
This therapy is expected to greatly improve the disease trajectory for many CF patients with at least one Phe508del mutation and this expectation should also apply to those groups with more advanced disease.
1. Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019;394(10212):1940-8.
2. Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, et al. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019;381(19):1809-19.