Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease with the Phe508del/minimal function genotype
| Autor: | L. Ferrillo, Fabiola De Gregorio, Vito Terlizzi, P. Medio, M. d'Ippolito, Giovanni Taccetti, S. Buonaurio, Carmela Colangelo, Donatello Salvatore, Giovanni Marsicovetere, Paola Iacotucci, Vincenzo Carnovale, Michela Francalanci, Michele D'Andria, Nicola Ferrara |
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| Rok vydání: | 2021 |
| Předmět: |
Adult
Male Pulmonary and Respiratory Medicine medicine.medical_specialty Indoles Pyrrolidines Adolescent Cystic Fibrosis Genotype Pyridines Quinolones Aminophenols Compound heterozygosity Gastroenterology Cystic fibrosis Ivacaftor Internal medicine medicine Humans Benzodioxoles Chloride Channel Agonists Retrospective Studies biology business.industry Retrospective cohort study Middle Aged medicine.disease Confidence interval Cystic fibrosis transmembrane conductance regulator Respiratory Function Tests Cohort biology.protein Pyrazoles Drug Therapy Combination Female business medicine.drug |
| Zdroj: | Respiratory Medicine. 189:106646 |
| ISSN: | 0954-6111 |
| DOI: | 10.1016/j.rmed.2021.106646 |
| Popis: | 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 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. |
| Databáze: | OpenAIRE |
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