Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease with the Phe508del/minimal function genotype - 08/12/21
, Paola Iacotucci a, 1, Vito Terlizzi b, 1, Carmela Colangelo c, Pietro Medio a, Lorenza Ferrillo a, Fabiola De Gregorio c, Michela Francalanci b, Giovanni Taccetti b, Serena Buonaurio a, Marcella d’Ippolito a, Giovanni Marsicovetere c, Michele D'Andria c, Nicola Ferrara a, Donatello Salvatore cAbstract |
Background |
Elexacaftor/tezacaftor/ivacaftor (E/T/I) is a cystic fibrosis transmembrane conductance regulator (CFTR) triple combination therapy used for the treatment of cystic fibrosis (CF) in patients aged ≥12 years who have at least one copy of the Phe508del mutation (F) in the CFTR gene or another mutation that is responsive to treatment with E/T/I. This study determined the effectiveness and safety of E/T/I treatment in a cohort of CF patients.
Methods |
This retrospective cohort study collected data from the first 6 months of treatment of patients with CF, compound heterozygotes for the F and a minimal function (MF) mutations, enrolled in an E/T/I compassionate use program only available to patients having ppFEV1<40 or who are considered for lung transplantation. Forty-seven patients were included. Follow-up was performed after 1, 3, and 6 months from the beginning of therapy, assessing lung function, body mass index (BMI), sweat chloride concentration (SCC), quality of life (QoL), and safety.
Results |
After 6 months of treatment, the mean (standard deviation (SD)) SCC decreased from 91.1 (19.3) mmol/L to 46.2 (24.2) mmol/L. The decrease of SCC was accompanied by improvement of lung function (mean (95% Confidence Interval (CI) absolute increase in ppFEV1 was 10.69 (8.05,13.33) after 1 month and 14.16 (11.43, 16.89) after 6 months of treatment), nutrition (mean (SD) BMI increased from 20.7 (3.0) kg/m2 at baseline to 22.6 (3.1) after 6 months), and QoL. No safety concerns were observed.
Conclusions |
E/T/I was clinically effective and safe in patients with advanced CF lung disease with an F/MF genotype.
Le texte complet de cet article est disponible en PDF.Highlights |
• | Advanced CF lung disease is partially reversible after treatment with E/T/I. |
• | E/T/I is safe and effective in severely affected patients with F/MF CFTR genotype. |
• | E/T/I decreases PEx and use of antibiotics in severe CF with F/MF CFTR genotype. |
Keywords : Cystic fibrosis, CFTR, Minimal function, Elexacaftor/tezacaftor/ivacaftor
Plan
Vol 189
Article 106646- novembre 2021 Retour au numéro
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