Cystic Fibrosis-related Diabetes — β-cell Function and Insulin Sensitivity in Patients With Cystic Fibrosis
Citation(s)
Adler AI, Shine BS, Chamnan P, Haworth CS, Bilton D Genetic determinants and epidemiology of cystic fibrosis-related diabetes: results from a British cohort of children and adults. Diabetes Care. 2008 Sep;31(9):1789-94. doi: 10.2337/dc08-0466. Epub 2008
Ali BR Is cystic fibrosis-related diabetes an apoptotic consequence of ER stress in pancreatic cells? Med Hypotheses. 2009 Jan;72(1):55-7. doi: 10.1016/j.mehy.2008.07.058. Epub 2008 Oct 11.
Association AD 1. Improving Care and Promoting Health in Populations: Standards of Medical Care in Diabetes-2020. Diabetes Care. 2020 Jan;43(Suppl 1):S7-S13. doi: 10.2337/dc20-S001. Review.
Austin A, Kalhan SC, Orenstein D, Nixon P, Arslanian S Roles of insulin resistance and beta-cell dysfunction in the pathogenesis of glucose intolerance in cystic fibrosis. J Clin Endocrinol Metab. 1994 Jul;79(1):80-5.
Haardt M, Benharouga M, Lechardeur D, Kartner N, Lukacs GL C-terminal truncations destabilize the cystic fibrosis transmembrane conductance regulator without impairing its biogenesis. A novel class of mutation. J Biol Chem. 1999 Jul 30;274(31):21873-7.
Hudson VM Rethinking cystic fibrosis pathology: the critical role of abnormal reduced glutathione (GSH) transport caused by CFTR mutation. Free Radic Biol Med. 2001 Jun 15;30(12):1440-61. Review.
Kelly A, Moran A Update on cystic fibrosis-related diabetes. J Cyst Fibros. 2013 Jul;12(4):318-31. doi: 10.1016/j.jcf.2013.02.008. Epub 2013 Apr 3. Review. Erratum in: J Cyst Fibros. 2014 Jan;13(1):119.
Koivula FNM, McClenaghan NH, Harper AGS, Kelly C Islet-intrinsic effects of CFTR mutation. Diabetologia. 2016 Jul;59(7):1350-1355. doi: 10.1007/s00125-016-3936-1. Epub 2016 Mar 31. Review. Erratum in: Diabetologia. 2017 Dec;60(12):2544.
Lanng S, Thorsteinsson B, Røder ME, Nerup J, Koch C Insulin sensitivity and insulin clearance in cystic fibrosis patients with normal and diabetic glucose tolerance. Clin Endocrinol (Oxf). 1994 Aug;41(2):217-23.
Mari A, Pacini G, Murphy E, Ludvik B, Nolan JJ A model-based method for assessing insulin sensitivity from the oral glucose tolerance test. Diabetes Care. 2001 Mar;24(3):539-48. Erratum in: Diabetes Care. 2014 Jul;37(7):2063.
Milla CE, Warwick WJ, Moran A Trends in pulmonary function in patients with cystic fibrosis correlate with the degree of glucose intolerance at baseline. Am J Respir Crit Care Med. 2000 Sep;162(3 Pt 1):891-5.
Moran A, Dunitz J, Nathan B, Saeed A, Holme B, Thomas W Cystic fibrosis-related diabetes: current trends in prevalence, incidence, and mortality. Diabetes Care. 2009 Sep;32(9):1626-31. doi: 10.2337/dc09-0586. Epub 2009 Jun 19.
Ode KL, Moran A New insights into cystic fibrosis-related diabetes in children. Lancet Diabetes Endocrinol. 2013 Sep;1(1):52-8. doi: 10.1016/S2213-8587(13)70015-9. Epub 2013 May 23. Review.
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.