PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy

• Published in: Journal of cystic fibrosis Vol. 20; no. 2; pp. 205 - 212
• Main Authors: Nichols, Dave P., Donaldson, Scott H., Frederick, Carla A., Freedman, Steven D., Gelfond, Daniel, Hoffman, Lucas R., Kelly, Andrea, Narkewicz, Michael R., Pittman, Jessica E., Ratjen, Felix, Sagel, Scott D., Rosenfeld, Margaret, Schwarzenberg, Sarah Jane, Singh, Pradeep K., Solomon, George M., Stalvey, Michael S., Kirby, Shannon, VanDalfsen, Jill M., Clancy, John P., Rowe, Steven M.
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier B.V 01.03.2021
• Subjects: Cftr; Chloride Channel Agonists - therapeutic use; Clinical trial; Cystic fibrosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Drug; Drug Combinations; Elexacaftor; Humans; Ivacaftor; Modulator; Observational Studies as Topic; Promise; Tezacaftor; Drug; Cftr; Promise; Modulator; Ivacaftor; Cystic fibrosis; Clinical trial; Elexacaftor; Tezacaftor
• ISSN: 1569-1993; 1873-5010
• DOI: 10.1016/j.jcf.2021.02.003

•PROMISE is a large clinical study to measure the impact of ETI by clinical use.•This observational study will measure outcomes over the first two years of drug use.•PROMISE includes eight sub-studies arranged and led by organ-system experts.•Several additional complementary studies around the globe will also study ETI.•Altogether, these studies will help determine future care and research priorities. 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.