Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting
| Autor: | Sylvaine Lefeuvre, Stéphane Dominique, S. Ramel, Quitterie Reynaud, Lydie Lemonnier, Clémence Dehillotte, Jinmi Baek, Dominique Hubert, Isabelle Danner Boucher, Prissile Bakouboula, Virginie Colomb-Jung, V. David, Anne Munck, Laurent Mely |
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| Rok vydání: | 2017 |
| Předmět: |
Pulmonary and Respiratory Medicine
Adult Male medicine.medical_specialty Staphylococcus aureus Adolescent Cystic Fibrosis Respiratory System Cystic Fibrosis Transmembrane Conductance Regulator Quinolones Azithromycin Aminophenols Cystic fibrosis Time Ivacaftor 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Internal medicine Medicine Humans 030212 general & internal medicine Intensive care medicine Adverse effect Child Chloride Channel Agonists business.industry Lumacaftor Retrospective cohort study medicine.disease Anti-Bacterial Agents Respiratory Function Tests Clinical trial 030228 respiratory system chemistry Pediatrics Perinatology and Child Health Pseudomonas aeruginosa Observational study Female France business medicine.drug |
| Zdroj: | Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 17(1) |
| ISSN: | 1873-5010 |
| Popis: | Background Ivacaftor has been shown to improve lung function and body weight in patients with CF and a gating mutation. Real-world evaluation is warranted to examine its safety and effectiveness over the long term. Methods A retrospective observational multicentre study collected clinical data in the year before and the 2years after ivacaftor initiation in patients with CF and a Gly551Asp- CFTR mutation. Results Fifty-seven patients were included. Mean absolute change in FEV 1 % predicted improved from baseline to Year 1 (8.4%; p Pseudomonas aeruginosa and Staphylococcus aureus positive cultures, and decreased IV antibiotics and maintenance treatment prescriptions (including azithromycin, Dornase alpha and nutritional supplements). No significant adverse events were reported. Conclusion The clinical benefits of ivacaftor reported in previous clinical trials were confirmed in a real-world setting two years post-initiation, also reducing treatment burden. |
| Databáze: | OpenAIRE |
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