ELX/TEZ/IVA
Elexacaftor-Tezacaftor-Ivacaftor in Cystic Fibrosis (F508del + Minimal Function)
Paziente / Popolazione Intervento / Esposizione Confronto Esito
In 403 patients ≥12 years with CF carrying one F508del and one minimal-function mutation, elexacaftor-tezacaftor-ivacaftor improved ppFEV1 and exacerbation rate compared with placebo.
N
403
Disegno
Multicentre phase-3 RCT
Esito
Absolute change in ppFEV1 at week 4 (key secondary: exacerbations at 24 weeks)
Rilevanza
1
RisultatoppFEV1 +13.8 percentage points (p<0.001); pulmonary exacerbations RR 0.37; sweat chloride -41.8 mmol/L.
Middleton PG, et al. N Engl J Med. 2019;381(19):1809-1819. 10.1056/NEJMoa1908639
Discussione e critica
Transformative — extends modulator eligibility to roughly 90% of patients with CF and is expected to add decades of median survival.