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Abstract #1359

Hyperpolarized Helium-3 MRI Detects the Effects of a Cftr Potentiator (Ivacaftor) Therapy in Subjects with Cystic Fibrosis and the G551D Mutation

Talissa A. Altes1, Mac Johnson2, John P. Mugler1, Deborah Froh3, Lucia Flors1, Grady Wilson Miller1, Jaime H. Mata1, Carlos L. Salinas1, Nick Tustison1, Po-Shun Lee2, Tao Song2, Karl Yen2, Eduard E. de Lange1, Martyn C. Botfield2

1Radiology, University of Virginia, Charlottesville, VA, United States; 2Vertex Pharmaceuticals Incorporated, Cambridge, MA, United States; 3Pediatric Pulmonology, University of Virginia, Charlottesville, VA, United States

This study to evaluate the ability of hyperpolarized helium-3 MR ventilation imaging to detect the effects of treatment with ivacaftor, a CFTR potentiator, demonstrated a rapid onset of improvement in lung ventilation with treatment and equally rapid return to near baseline ventilation following cessation of treatment. Eight subjects with CF were imaged on 5 different days, 2 weeks apart (Days 0, 14, 28, 42, and 56), and were treated with Ivacaftor from Days 14 to 42. Interim data analysis has been conducted on the first 4 subjects in the study, although all 8 subjects have completed the trial.