Cystic Fibrosis–Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes

Melissa S. Putman, Andrew W. Norris, Rebecca L. Hull, Michael R. Rickels, Lori Sussel, Scott M. Blackman, Christine L. Chan, Katie Larson Ode, Tanicia Daley, Arlene A. Stecenko, Antoinette Moran, Meagan J. Helmick, Sharon Cray, Jessica A. Alvarez, Virginia A. Stallings, Katherine L. Tuggle, John P. Clancy, Thomas L. Eggerman, John F. Engelhardt, Andrea Kelly

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)677-689
Number of pages13
Issue number6
StatePublished - Jun 2023

Bibliographical note

Funding Information:
M.R.R. and A.K. are supported in part by Public Health Service Research Grant R01 DK97830. A.K. is supported by grants from the Cystic Fibrosis Foundation. A.W.N. and J.F.E. are supported in part by grants R01 DK115791, RC2 DK124207, and P30 DK054759. T.D., A.A.S., and J.A.A. are supported in part by National Institutes of Health (NIH) grant P30 DK125013. M.S.P. is supported by R01 DK119699 and grants from the Cystic Fibrosis Foundation. A.M. is supported by NIH grant R01 DK101402. J.A.A. is supported by NIH grant R01 DK133523, Cystic Fibrosis Foundation grant ALVARE19A0, and Cystic Fibrosis Foundation grant ALVARE22A0. K.L.O. is supported in part by P30 DK054759 and grants from the Cystic Fibrosis Foundation.

Publisher Copyright:
© 2023 by the American Diabetes Association.

PubMed: MeSH publication types

  • Journal Article
  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural
  • Review


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