Dominique Hubert1, Clémence Dehillotte2, Anne Munck3, Valérie David4, Jinmi Baek5, Laurent Mely6, Stéphane Dominique7, Sophie Ramel8, Isabelle Danner Boucher9, Sylvaine Lefeuvre10, Quitterie Reynaud11, Virginie Colomb-Jung2, Prissile Bakouboula5, Lydie Lemonnier2. 1. Pulmonary Department, Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; Université Paris Descartes, Sorbonne Paris Cité, Paris, France. Electronic address: dominique.hubert@aphp.fr. 2. Vaincre la Mucoviscidose, Paris, France. 3. Pediatric CF Centre, Robert Debré Hospital, AP-HP, Paris, France. 4. Pediatric CF Centre, Hôpital Mère-Enfant, Nantes, France. 5. Clinical Research Unit, Cochin Hospital, AP-HP, Paris, France. 6. CF Centre, Renée Sabran Hospital, Giens, France. 7. Pulmonary Department, Adult CF Centre, Charles Nicolle Hospital, Rouen University Hospital, Rouen, France. 8. CF Centre, Centre héliomarin de Perharidy, Roscoff, France. 9. Pulmonary Department, Adult CF Centre, Laennec Hospital, Nantes, France. 10. Paediatric CF Centre, Hôpital Sud, Rennes, France. 11. Adult CF Centre Lyon Sud, Hospices Civils de Lyon, Lyon, France.
Abstract
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 FEV1% predicted improved from baseline to Year 1 (8.4%; p<0.001) and Year 2 (7.2%; p=0.006). Statistically significant benefits were observed with increased body mass index, fewer 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.
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 FEV1% predicted improved from baseline to Year 1 (8.4%; p<0.001) and Year 2 (7.2%; p=0.006). Statistically significant benefits were observed with increased body mass index, fewer 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.
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