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Advances in Medicine
Volume 2014, Article ID 947923, 5 pages
http://dx.doi.org/10.1155/2014/947923
Research Article

Ivacaftor Therapy in CF Patients: Single Center Experience

1Department of Pediatrics, University of Florida, 1600 SW Archer Road, Suite HD 604, P.O. Box 100296, Gainesville, FL 32610, USA
2Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Florida, 1600 SW Archer Road, P.O. Box 100225, Gainesville, FL 32610, USA
3Division of Pediatric Pulmonary Medicine, Department of Pediatrics, University of Florida, 1600 SW Archer Road, P.O. Box 100296, Gainesville, FL 32610, USA

Received 8 May 2014; Revised 3 September 2014; Accepted 15 September 2014; Published 22 October 2014

Academic Editor: Gernot Zissel

Copyright © 2014 Pritish Mondal et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

Ivacaftor is the first novel cystic fibrosis pharmaceutical that acts at the molecular level to potentiate cystic fibrosis transmembrane conductance regulator (CFTR) function and was first approved for clinical use in 2012. We are sharing our single center experience of five patients: four from pediatric age group and one adult patient. All patients had both subjective and objective improvements in their health. Despite established lung disease, our patients had significant improvement in both their FEV1 (forced expiratory volume in 1 second) and FE and BMI (body mass index). Larger studies demonstrated only 6.7% improvement in mean FEV1 after starting Ivacaftor therapy but their patient population had normal lung function to begin with. In contrast our case series demonstrates that, in patients with established lung disease and diminished lung function, Ivacaftor can be expected to result in much higher recovery in lung function. Mean FEV1 improved by 35% in our case series. Ivacaftor is extremely expensive, costing $300,000 per patient per year requiring lifelong therapy, hence requiring prior authorizations from most third-party payers in the USA. The knowledge shared from our experience will be useful for other clinicians to petition healthcare policymakers on behalf of their patients.