Laura Kirwan1, Godfrey Fletcher1, Mary Harrington1, Paulina Jeleniewska1, Shijun Zhou1, Brian Casserly2, Charles G Gallagher3, Peter Greally4, Cedric Gunaratnam5, Mary Herzig6, Barry Linnane7,8, Noel Gerard McElvaney5, Edward F McKone3, Paul McNally9, David Mullane10, Muireann Ní Chróinín10, Michael O'Mahony11, Barry J Plant12, Abaigeal D Jackson1. 1. 1 Cystic Fibrosis Registry of Ireland, Woodview House, University College Dublin, Belfield, Dublin, Ireland. 2. 2 University Hospital Limerick, Dooradoyle, Limerick, Ireland. 3. 3 National Referral Centre for Adult Cystic Fibrosis, St. Vincent's University Hospital, Dublin, Ireland. 4. 4 Children's Hospital Group, Rialto, Dublin, Ireland. 5. 5 Cystic Fibrosis Unit, Beaumont Hospital, Dublin, Ireland. 6. 6 Paediatrics Department and. 7. 7 Graduate Entry Medical School and Centre for Interventions in Infection, Inflammation, and Immunity, University of Limerick, Limerick, Ireland. 8. 8 National Children's Research Centre, Crumlin, Dublin, Ireland. 9. 9 Royal College of Surgeons in Ireland, Our Lady's Children's Hospital, Crumlin, Dublin, Ireland; and. 10. 10 Cork Paediatric Cystic Fibrosis Centre and. 11. 11 Respiratory Department, University Hospital Galway, Galway, Ireland. 12. 12 Cork Adult Cystic Fibrosis Centre, University College Cork, Cork University Hospital, Cork, Ireland.
Abstract
RATIONALE: Patient registries have the potential to collect and analyze high-quality postauthorization data on new medicines. OBJECTIVES: We used cystic fibrosis (CF) registry data to assess outcomes after the initiation of ivacaftor, a CF transmembrane conductance regulator (CFTR) potentiator approved for the treatment of CF with a defective gating CFTR mutation. METHODS: Longitudinal trends were examined using mixed-effects regression analysis in 80 ivacaftor-treated patients with CF aged 6 to 56 years registered with the CF Registry of Ireland with at least 36 months of before and after commencement data. The effects of ivacaftor treatment on forced expiratory volume in 1 second (FEV1) % predicted, body mass index (BMI), hospitalization for pulmonary exacerbation, and oral and intravenous antibiotic use were assessed. RESULTS: In the 36 months after ivacaftor initiation, FEV1% predicted improved by 2.26% per annum (95% confidence interval [CI], 0.2 to 4.3) for patients aged younger than 12 years, remained unchanged for 12- to younger than 18-year-olds (95% CI, -1.9 to 2.9), and declined in adults by 1.74% per annum (95% CI, -3.1 to -0.4). BMI in adults increased 0.28 kg/m2 per annum (95% CI, 0.03 to 0.5), and there was no significant change in BMI z-score in children (95% CI, -0.01 to 0.1). In the year after ivacaftor initiation, intravenous antibiotic treatment reduced by 46% (95% CI, -62.5% to -23.3%, oral antibiotic treatment reduced by 49% (95% CI, -61.1% to -32.1%), and there was no significant reduction in hospitalization (95% CI, -59.2% to 9.7%). CONCLUSIONS: In this study of real-world CF registry data, clinical outcomes improved and healthcare resource utilization decreased after commencing ivacaftor.
RATIONALE: Patient registries have the potential to collect and analyze high-quality postauthorization data on new medicines. OBJECTIVES: We used cystic fibrosis (CF) registry data to assess outcomes after the initiation of ivacaftor, a CF transmembrane conductance regulator (CFTR) potentiator approved for the treatment of CF with a defective gating CFTR mutation. METHODS: Longitudinal trends were examined using mixed-effects regression analysis in 80 ivacaftor-treated patients with CF aged 6 to 56 years registered with the CF Registry of Ireland with at least 36 months of before and after commencement data. The effects of ivacaftor treatment on forced expiratory volume in 1 second (FEV1) % predicted, body mass index (BMI), hospitalization for pulmonary exacerbation, and oral and intravenous antibiotic use were assessed. RESULTS: In the 36 months after ivacaftor initiation, FEV1% predicted improved by 2.26% per annum (95% confidence interval [CI], 0.2 to 4.3) for patients aged younger than 12 years, remained unchanged for 12- to younger than 18-year-olds (95% CI, -1.9 to 2.9), and declined in adults by 1.74% per annum (95% CI, -3.1 to -0.4). BMI in adults increased 0.28 kg/m2 per annum (95% CI, 0.03 to 0.5), and there was no significant change in BMI z-score in children (95% CI, -0.01 to 0.1). In the year after ivacaftor initiation, intravenous antibiotic treatment reduced by 46% (95% CI, -62.5% to -23.3%, oral antibiotic treatment reduced by 49% (95% CI, -61.1% to -32.1%), and there was no significant reduction in hospitalization (95% CI, -59.2% to 9.7%). CONCLUSIONS: In this study of real-world CF registry data, clinical outcomes improved and healthcare resource utilization decreased after commencing ivacaftor.
Authors: Anh-Thu N Lam; Melis A Aksit; Briana Vecchio-Pagan; Celeste A Shelton; Derek L Osorio; Arianna F Anzmann; Loyal A Goff; David C Whitcomb; Scott M Blackman; Garry R Cutting Journal: J Clin Invest Date: 2020-01-02 Impact factor: 14.808
Authors: Guido Veit; Dillon F Da Fonte; Radu G Avramescu; Aiswarya Premchandar; Miklos Bagdany; Haijin Xu; Dennis Bensinger; Daniel Stubba; Boris Schmidt; Elias Matouk; Gergely L Lukacs Journal: J Cyst Fibros Date: 2019-10-31 Impact factor: 5.482
Authors: Guido Veit; Ariel Roldan; Mark A Hancock; Dillon F Da Fonte; Haijin Xu; Maytham Hussein; Saul Frenkiel; Elias Matouk; Tony Velkov; Gergely L Lukacs Journal: JCI Insight Date: 2020-09-17