Asunto(s)
Enfermedades Autoinmunes del Sistema Nervioso/tratamiento farmacológico , Interferones/metabolismo , Malformaciones del Sistema Nervioso/tratamiento farmacológico , Inhibidores de la Transcriptasa Inversa/uso terapéutico , Enfermedades Autoinmunes del Sistema Nervioso/metabolismo , Circulación Cerebrovascular/efectos de los fármacos , Didesoxinucleósidos/uso terapéutico , Combinación de Medicamentos , Francia , Expresión Génica/efectos de los fármacos , Humanos , Interferones/genética , Lamivudine/uso terapéutico , Malformaciones del Sistema Nervioso/metabolismo , Proyectos Piloto , Zidovudina/uso terapéuticoRESUMEN
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.