Patrick A Flume1, Claire E Wainwright2, D Elizabeth Tullis3, Sally Rodriguez4, Minoo Niknian5, Mark Higgins6, Jane C Davies7, Jeffrey S Wagener8. 1. Departments of Medicine and Pediatrics, Medical University of South Carolina, 96 Jonathan Lucas St, Room 812-CSB, MSC 630, Charleston, SC 29425, USA. Electronic address: flumepa@musc.edu. 2. University of Queensland, Level 7, Centre for Child Health Research, Graham St, South Brisbane, Queensland 4101, Australia; Lady Cilento Children's Hospital, 501 Stanley St, South Brisbane 4101, Australia. Electronic address: claire.wainwright@health.qld.gov.au. 3. Division of Respirology, Keenan Research Centre of Li Ka Shing Knowledge Institute, Department of Medicine, St. Michael's Hospital, University of Toronto, 1 King's College Circle, 6263 Medical Sciences Building, Toronto, ON M5S 1A8, Canada. Electronic address: tullis@smh.ca. 4. Johnson & Johnson Medical Devices, 325 Paramount Dr, Raynham, MA 02767, USA. Electronic address: srodr127@its.jnj.com. 5. Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA 02210, USA. Electronic address: minoo_niknian@vrtx.com. 6. Vertex Pharmaceuticals (Europe) Limited, 86-88 Jubilee Avenue, Milton Park, Abingdon, Oxfordshire OX14 4RW, UK. Electronic address: mark_higgins@vrtx.com. 7. National Heart and Lung Institute, Imperial College, London, UK; Department of Pediatric Respiratory Medicine, Royal Brompton and Harefield National Health Service Foundation Trust, Sydney Street, London SW3 6NP, UK. Electronic address: j.c.davies@imperial.ac.uk. 8. Department of Pediatrics, University of Colorado School of Medicine, 13123 E 16th Ave, Aurora, CO 80045, USA. Electronic address: jeffrey.wagener@ucdenver.edu.
Abstract
BACKGROUND: Pulmonary exacerbations (PEx) are associated with acute loss of lung function that is often not recovered after treatment. We investigated lung function recovery following PEx for ivacaftor- and placebo-treated subjects. METHODS: Short- and long-term pulmonary function recovery data after PEx were summarized from a placebo-controlled trial in 161 cystic fibrosis patients≥12years old with the G551D-CFTR mutation (NCT00909532). Short-term recovery was measured 2 to 8weeks after treatment, and long-term recovery was determined at the end-of-study, both compared with baseline measured just prior to the PEx. RESULTS: Fewer patients receiving ivacaftor experienced a PEx than patients receiving placebo (33.7% vs. 56.4%; P=0.004) and had a lower adjusted incidence rate of PEx (0.589 vs. 1.382; P<0.001). The proportion of PEx followed by full short-term recovery of percent predicted forced expiratory volume in 1s was similar (ivacaftor vs. placebo, 57.1% vs. 53.7), as was the proportion of patients having long-term recovery (46.4% vs. 47.7%). CONCLUSIONS:Ivacaftor treatment reduces the frequency of PEx but does not improve on the rate of complete lung function recovery after PEx when compared with placebo.
RCT Entities:
BACKGROUND: Pulmonary exacerbations (PEx) are associated with acute loss of lung function that is often not recovered after treatment. We investigated lung function recovery following PEx for ivacaftor- and placebo-treated subjects. METHODS: Short- and long-term pulmonary function recovery data after PEx were summarized from a placebo-controlled trial in 161 cystic fibrosispatients≥12years old with the G551D-CFTR mutation (NCT00909532). Short-term recovery was measured 2 to 8weeks after treatment, and long-term recovery was determined at the end-of-study, both compared with baseline measured just prior to the PEx. RESULTS: Fewer patients receiving ivacaftor experienced a PEx than patients receiving placebo (33.7% vs. 56.4%; P=0.004) and had a lower adjusted incidence rate of PEx (0.589 vs. 1.382; P<0.001). The proportion of PEx followed by full short-term recovery of percent predicted forced expiratory volume in 1s was similar (ivacaftor vs. placebo, 57.1% vs. 53.7), as was the proportion of patients having long-term recovery (46.4% vs. 47.7%). CONCLUSIONS:Ivacaftor treatment reduces the frequency of PEx but does not improve on the rate of complete lung function recovery after PEx when compared with placebo.
Authors: Vin Tangpricha; Joshua Lukemire; Yuqing Chen; José Nilo G Binongo; Suzanne E Judd; Ellen S Michalski; Moon J Lee; Seth Walker; Thomas R Ziegler; Rabin Tirouvanziam; Susu M Zughaier; Supavit Chesdachai; Wendy A Hermes; James F Chmiel; Ruth E Grossmann; Amit Gaggar; Patricia M Joseph; Jessica A Alvarez Journal: Am J Clin Nutr Date: 2019-03-01 Impact factor: 7.045
Authors: Christopher H Goss; Sonya L Heltshe; Natalie E West; Michelle Skalland; Don B Sanders; Raksha Jain; Tara L Barto; Barbra Fogarty; Bruce C Marshall; Donald R VanDevanter; Patrick A Flume Journal: Am J Respir Crit Care Med Date: 2021-12-01 Impact factor: 21.405