Journal article

Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis

Patrick O Hanafin, Isabelle Sermet-Gaudelus, Matthias Griese, Matthias Kappler, Helmut Ellemunter, Carsten Schwarz, John Wilson, Marsha Tan, Tony Velkov, Gauri G Rao, Elena K Schneider-Futschik



Background: The advent of cystic fibrosis transmembrane conductance regulator protein (CFTR) modulators like ivacaftor have revolutionised the treatment of cystic fibrosis (CF). However, due to the plethora of variances in disease manifestations in CF, there are inherent challenges in unified responses under CFTR modulator treatment arising from variability in patient outcomes. The pharmacokinetic (PK) data available for ivacaftor-lumacaftor cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator drug combination is limited. Methods: Secondary objectives were to identify (1) patient characteristics and (2) the interactions between ivacaftor-lumacaftor responsible for interi..

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Awarded by University of Melbourne

Funding Acknowledgements

ES-F supported by a research grant (APP1157287) from The University of Melbourne and the Australian National Health and Medical Research Council (NHMRC) as Biomedical Research Fellow. Portions of this work were supported by the Peter Phelan grant from the Thoracic Society of Australia and New Zealand. The content is solely the responsibility of the authors and does not necessarily represent the official views of the Australian National Health and Medical Research Council.