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PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy
Authors:Dave P. Nichols  Scott H. Donaldson  Carla A. Frederick  Steven D. Freedman  Daniel Gelfond  Lucas R. Hoffman  Andrea Kelly  Michael R. Narkewicz  Jessica E. Pittman  Felix Ratjen  Scott D. Sagel  Margaret Rosenfeld  Sarah Jane Schwarzenberg  Pradeep K. Singh  George M. Solomon  Michael S. Stalvey  Shannon Kirby  Jill M. VanDalfsen  Steven M. Rowe
Affiliation:1. Seattle Children''s Research Institute, Department of Pediatrics, University of Washington, USA;2. Department of Medicine, Univ. North Carolina at Chapel Hill, USA;3. Jacobs School of Medicine and Biomedical Sciences of the University of Buffalo, USA;4. Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA;5. Western New York Pediatric Gastroenterology, USA;6. Seattle Children''s Research Institute, Department of Pediatrics and Microbiology, University of Washington, USA;7. Department of Endocrinology, University of Pennsylvania, USA;8. Digestive Health Institute, Children''s Hospital Colorado and University of Colorado School of Medicine, Aurora, CO, USA;9. Department of Pediatrics, Washington University School of Medicine, USA;10. Department of Pediatrics, University of Toronto, USA;11. Seattle Children''s Research Institute, Department of Pediatrics and Epidemiology, University of Washington, USA;12. Department of Pediatrics, Children''s Hospital Colorado and University of Colorado School of Medicine, Aurora, CO, USA;13. University of Minnesota Masonic Children''s Hospital, Minneapolis, MN, USA;14. Department of Microbiology, University of Washington, USA;15. Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, USA;p. Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, USA;q. Therapeutics Development Network Coordinating Center, Seattle Children''s Hospital, USA;r. Cystic Fibrosis Foundation, USA;s. Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, USA
Abstract:Highly effective CFTR modulator drug therapy is increasingly available to those with cystic fibrosis. Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term outcomes, patient care needs, and future research priorities. PROMISE is a large, multi-disciplinary academic study focused on the broad impacts of starting elexacaftor/tezacaftor/ivacaftor in the US population age 6 years and older. The many areas of investigation and rationale for each are discussed by organ systems, along with recognition of remaining important questions that will not be addressed by this study alone. Knowledge gained through this and multiple complementary studies around the world will help to understand important health outcomes, clinical care priorities, and research needs for a large majority of people treated with these or similarly effective medications targeting the primary cellular impairment in cystic fibrosis.
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