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

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; John P Clancy; Steven M Rowe

doi:10.1016/j.jcf.2021.02.003

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

J Cyst Fibros. 2021 Mar;20(2):205-212. doi: 10.1016/j.jcf.2021.02.003. Epub 2021 Feb 19.

Authors

Dave P Nichols¹, Scott H Donaldson², Carla A Frederick³, Steven D Freedman⁴, Daniel Gelfond⁵, Lucas R Hoffman⁶, Andrea Kelly⁷, Michael R Narkewicz⁸, Jessica E Pittman⁹, Felix Ratjen¹⁰, Scott D Sagel¹¹, Margaret Rosenfeld¹², Sarah Jane Schwarzenberg¹³, Pradeep K Singh¹⁴, George M Solomon¹⁵, Michael S Stalvey¹⁵, Shannon Kirby¹⁶, Jill M VanDalfsen¹⁶, John P Clancy¹⁷, Steven M Rowe¹⁵

Affiliations

¹ Seattle Children's Research Institute, Department of Pediatrics, University of Washington, USA. Electronic address: [email protected].
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.
¹⁶ Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, USA.
¹⁷ Cystic Fibrosis Foundation, 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.

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

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Chloride Channel Agonists / therapeutic use*
Cystic Fibrosis / drug therapy*
Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
Drug Combinations
Humans
Observational Studies as Topic

Substances

Chloride Channel Agonists
Drug Combinations
Cystic Fibrosis Transmembrane Conductance Regulator

Abstract

Publication types

MeSH terms

Substances

Grants and funding