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

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.

Keywords: Cftr; Clinical trial; Cystic fibrosis; Drug; Elexacaftor; Ivacaftor; Modulator; Promise; Tezacaftor.

Figure 1.

Organ systems being evaluated in the PROMISE study. A wide range of organ systems and disease manifestations are being studied during the multi-year observational period. This will allow us to better understand the impacts of ETI therapy in clinical use and where it may be most beneficial to focus or prioritize ongoing therapeutic and research goals in those treated with highly effective modulator drugs.