Real‐life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients

• DOI: 10.1002/prp2.1015
• Publication Date: 2022-11-28T11:24:02Z
• Authors: Renate Kos; Anne H. Neerincx; Dominic W. Fenn; Paul Brinkman; Rianne Lub; Steffie E. M. Vonk; Jolt Roukema; Monique H. Reijers; Suzanne W. J. Ter...; Josje Altenburg; Christof J. Majoor; Lieuwe D. Bos; Eric G. Haarman; Anke H. Maitland‐...
• Keywords: Ivacaftor

Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in CF patients (≥6 years) with ≥1 Phe508del mutation and minimal function mutation. In October 2019, ETI became available on compassionate use basis for Dutch severe lung disease. Our objective was to investigate safety of this patient group real-life setting. A multicenter longitudinal observational study conducted examine changes FEV1 , BMI, adverse events at initiation 1, 3, 6, 12 months after starting ETI. The number exacerbations recorded the before treatment. Patients eligible had <40% predicted. Wilcoxon signed-rank test analyzed over time. Twenty subjects were included followed up Treatment well tolerated mild side effects reported, namely, rash (15%) stomach ache (20%) 80% resolving within 1 month. Mean absolute increase 11.8/13.7% (p ≤ .001) BMI 0.49/1.87 kg/m2 < .001-0.02) 1/12 months, respectively. comparison pretrial, there marked reduction initiation. findings show long-term treatment disease associated increased less exacerbations, only effects.