Journal article
Cystic Fibrosis-Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes
Diabetes care, Vol.46(6), pp.1112-1123
06/2023
DOI: 10.2337/dc23-0380
PMCID: PMC10234745
PMID: 37125948
Abstract
Cystic fibrosis (CF) is a recessive disorder arising from mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CFTR is expressed in numerous tissues, with high expression in the airways, small and large intestine, pancreatic and hepatobiliary ducts, and male reproductive tract. CFTR loss in these tissues disrupts regulation of salt, bicarbonate, and water balance across their epithelia, resulting in a systemic disorder with progressive organ dysfunction and damage. Pancreatic exocrine damage ultimately manifests as pancreatic exocrine insufficiency that begins as early as infancy. Pancreatic remodeling accompanies this early damage, during which abnormal glucose tolerance can be observed in toddlers. With increasing age, however, insulin secretion defects progress such that CF-related diabetes (CFRD) occurs in 20% of teens and up to half of adults with CF. The relevance of CFRD is highlighted by its association with increased morbidity, mortality, and patient burden. While clinical research on CFRD has greatly assisted in the care of individuals with CFRD, key knowledge gaps on CFRD pathogenesis remain. Furthermore, the wide use of CFTR modulators to restore CFTR activity is changing the CFRD clinical landscape and the field's understanding of CFRD pathogenesis. For these reasons, the National Institute of Diabetes and Digestive and Kidney Diseases and the Cystic Fibrosis Foundation sponsored a CFRD Scientific Workshop, 23-25 June 2021, to define knowledge gaps and needed research areas. This article describes the findings from this workshop and plots a path for CFRD research that is needed over the next decade.
Details
- Title: Subtitle
- Cystic Fibrosis-Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes
- Creators
- Melissa S Putman - Massachusetts General HospitalAndrew W Norris - University of IowaRebecca L Hull - VA Puget Sound Health Care SystemMichael R Rickels - University of PennsylvaniaLori Sussel - University of Colorado Anschutz Medical CampusScott M Blackman - Johns Hopkins MedicineChristine L Chan - University of Colorado Anschutz Medical CampusKatie Larson Ode - University of IowaTanicia Daley - Emory UniversityArlene A Stecenko - Emory UniversityAntoinette Moran - University of MinnesotaMeagan J Helmick - Cystic Fibrosis FoundationSharon Cray - Cystic Fibrosis FoundationJessica A Alvarez - Emory UniversityVirginia A Stallings - Children's Hospital of PhiladelphiaKatherine L Tuggle - Cystic Fibrosis FoundationJohn P Clancy - Cystic Fibrosis FoundationThomas L Eggerman - National Institute of Diabetes and Digestive and Kidney DiseasesJohn F Engelhardt - University of IowaAndrea Kelly - Children's Hospital of Philadelphia
- Resource Type
- Journal article
- Publication Details
- Diabetes care, Vol.46(6), pp.1112-1123
- DOI
- 10.2337/dc23-0380
- PMID
- 37125948
- PMCID
- PMC10234745
- NLM abbreviation
- Diabetes Care
- eISSN
- 1935-5548
- Grant note
- name: CFF; name: NIH, award: P30DK125013, R01 DK133523, R01DK101402; name: AAS; DOI: 10.13039/100000897, name: CF Foundation, award: P30 DK054759, R01 DK115791, R01DK119699, RC2 DK124207; name: Public Health Service, award: R01 DK97830
- Language
- English
- Electronic publication date
- 05/01/2023
- Date published
- 06/2023
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Endocrinology and Diabetes; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Radiation Oncology; Biochemistry and Molecular Biology; Internal Medicine
- Record Identifier
- 9984400760002771
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