Donatello Salvatore1, Vito Terlizzi2, Michela Francalanci2, Giovanni Taccetti2, Barbara Messore3, Carlotta Biglia3, Giovanna Pisi4, Maria Adelaide Calderazzo5, Mimma Caloiero5, Giovanna Pizzamiglio6, Fabio Majo7, Federico Cresta8, Giuseppina Leonetti9, Domenica De Venuto9. 1. Cystic Fibrosis Center, AOR Ospedale San Carlo, Potenza, Italy. Electronic address: fibrosicistica@ospedalesancarlo.it. 2. Cystic Fibrosis Center, Department of Pediatric Medicine, Anna Meyer Children's Hospital, Florence, Italy. 3. Adult Cystic Fibrosis Center, Azienda Ospedaliera Universitaria San Luigi Gonzaga, Orbassano, Italy. 4. Cystic Fibrosis Unit, Department of Pediatrics, University Hospital, Parma, Italy. 5. Cystic Fibrosis Center, Hospital Giovanni Paolo II, Lamezia Terme, Italy. 6. Cystic Fibrosis Center - Adult Unit, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. 7. Cystic Fibrosis Unit, Bambino Gesù Children's Hospital, Rome, Italy. 8. Cystic Fibrosis Center, IRCCS G. Gaslini Institute, Genoa, Italy. 9. Cystic Fibrosis Regional Center, Department of Biomedical and Human Oncology, Pediatrics Section, U.O. "B. Trambusti", Policlinico, University of Bari, Bari, Italy.
Abstract
BACKGROUND: Ivacaftor is an innovative treatment for CF. Ivacaftor monotherapy in a phase III trial for patients with F508del and a residual function (RF) mutation showed improvement in lung function. We evaluated the effectiveness and safety of ivacaftor in patients with severe CF carrying RF mutations. METHODS: Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use program. Data were collected 1 year before and 1 year after commencement of ivacaftor. RESULTS: Twenty-six patients received ivacaftor. The mean [standard deviation (SD)] percent predicted FEV1 significantly increased from 33.9% (8.3) before treatment to 44.0% (10.7) after 12 months of treatment (p < 0.00001). The mean distance in the 6-min walking-test significantly improved from 458.2 (110.5) m at baseline to 524.8 (91.9) m after 12 months (p < 0.00001). The overall number of days of antibiotic therapy decreased from 1693 during the year before ivacaftor to 714 in the year following ivacaftor, and the number of days of intravenous antibiotic treatment dropped from 714 to 88; both results were statistically significant (p < 0.00001). Patients needing intravenous therapy decreased from 23 to 5 of 26. The mean (SD) sweat chloride level decreased from a baseline of 79 (22.3) mmol/L to 65 (30.6) mmol/L, but this variation was not significant (p = 0.26). No safety concerns were registered. CONCLUSIONS: In patients with CFTR mutations that confer RF with severe lung disease, treatment with Ivacaftor is safe and results in a clinically significant improvement that was evident at 1 month and maintained at 12 months.
BACKGROUND:Ivacaftor is an innovative treatment for CF. Ivacaftor monotherapy in a phase III trial for patients with F508del and a residual function (RF) mutation showed improvement in lung function. We evaluated the effectiveness and safety of ivacaftor in patients with severe CF carrying RF mutations. METHODS: Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use program. Data were collected 1 year before and 1 year after commencement of ivacaftor. RESULTS: Twenty-six patients received ivacaftor. The mean [standard deviation (SD)] percent predicted FEV1 significantly increased from 33.9% (8.3) before treatment to 44.0% (10.7) after 12 months of treatment (p < 0.00001). The mean distance in the 6-min walking-test significantly improved from 458.2 (110.5) m at baseline to 524.8 (91.9) m after 12 months (p < 0.00001). The overall number of days of antibiotic therapy decreased from 1693 during the year before ivacaftor to 714 in the year following ivacaftor, and the number of days of intravenous antibiotic treatment dropped from 714 to 88; both results were statistically significant (p < 0.00001). Patients needing intravenous therapy decreased from 23 to 5 of 26. The mean (SD) sweat chloride level decreased from a baseline of 79 (22.3) mmol/L to 65 (30.6) mmol/L, but this variation was not significant (p = 0.26). No safety concerns were registered. CONCLUSIONS: In patients with CFTR mutations that confer RF with severe lung disease, treatment with Ivacaftor is safe and results in a clinically significant improvement that was evident at 1 month and maintained at 12 months.
Authors: Fernanda Dos Anjos; Jonatha Wruck; Thiago Inácio Teixeira do Carmo; Victor Emanuel Miranda Soares; Débora Tavares de Resende E Silva; Margarete Dulce Bagatini; Sarah Franco Vieira de Oliveira Maciel Journal: Clin Med Res Date: 2022-02-07
Authors: Vito Terlizzi; Rita Padoan; Laura Claut; Carla Colombo; Benedetta Fabrizzi; Marco Lucarelli; Sabina Maria Bruno; Alice Castaldo; Paolo Bonomi; Giovanni Taccetti; Antonella Tosco Journal: Diagnostics (Basel) Date: 2020-12-12