Chest
Original ResearchGenetic And Developmental DisordersEffects of Ivacaftor in Patients With Cystic Fibrosis Who Carry the G551D Mutation and Have Severe Lung Disease
Section snippets
Materials and Methods
We conducted a retrospective case-control study of patients receiving ivacaftor on the compassionate use program in the United Kingdom and Ireland. All centers with adult patients enrolled into this program were contacted to participate in the study.
Results
Data were collected on 21 patients with severe CF lung disease who received ivacaftor (composing 84% of the eligible adult patients in the United Kingdom and Ireland receiving ivacaftor under the compassionate use program) and 35 matched control subjects who did not carry the G551D mutation. Follow-up data were available for a median of 237 days (range, 125-270 days) after ivacaftor commencement.
Baseline demographics of the study population are contained in Table 1 and e-Table 2. Absolute
Discussion
In patients with CF who carry the G551D mutation and have severe pulmonary disease, provision of ivacaftor led to significant improvements in FEV1 and FVC and improvements in weight and BMI. In addition, we have also shown a significant and clinically important reduction in the number of IV treatment days.
The clinical efficacy of ivacaftor in patients with CF who carry the G551D mutation and have mild to moderate pulmonary disease has been established in two large, phase 3 clinical trials.6, 7
Acknowledgments
Author contributions: A. R. H. had full access to all of the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis. P. J. B. served as principal author. P. J. B., B. J. P., S. B., N. J. S., N. J. B., N. T. S., T. D., A. M. J., and A. R. H. contributed to the study design; P. J. B., B. J. P., A. N., S. B., N. J. S., N. J. B., N. T. S., T. D., S. S., I. F., C. G., and A. R. H. contributed to data acquisition; P. J. B. and A. R. H.
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FUNDING/SUPPORT: This study was supported by the Manchester Adult Cystic Fibrosis Centre. Dr Horsley is funded by a National Institute for Health Research Clinician Scientist award [NIHR CS012-013].
Some of the data included in this manuscript have been published in abstract form (Barry P, Plant B, Nair A, et al. J Cyst Fibros. 2013;12[1]:S15 and Barry P, Plant B, Simmonds NJ, et al. J Cyst Fibros. 2013;12[1]:S62) and presented at the European Cystic Fibrosis Conference, June 12-15, 2013, Lisbon, Portugal, and at the North American Cystic Fibrosis Conference, October 17-19, 2013, Salt Lake City, UT.
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