Endocrine Complications of Cystic Fibrosis

Kelly, Andrea; Marks, Brynn E.; Stalvey, Michael S.

doi:10.1016/j.ccm.2022.06.013

Cited by 4 publications

(8 citation statements)

References 121 publications

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“…85 EPI is extremely common in CF, and ~85% of pwCF are diagnosed with EPI. 4 The deficiency of lipase secretion with EPI prevents hydrolysis and, thus, absorption of lipid. In addition to the classic signs and symptoms associated with fat malabsorption, faltering weight in infants and children and unexplained weight loss in adults may also be attributed to EPI.…”

Section: Pancreasmentioning

confidence: 99%

“…CF can compromise pancreas function, preventing adequate secretion of pancreatic enzymes 84 . EPI is defined as “the condition that exocrine pancreas secretes pancreatic enzymes, that is, lipase, amylase, or proteases lower than normal levels.” 85 EPI is extremely common in CF, and ~85% of pwCF are diagnosed with EPI 4 . The deficiency of lipase secretion with EPI prevents hydrolysis and, thus, absorption of lipid.…”

Section: Pathophysiology Of the Cf Gi Tractmentioning

confidence: 99%

“…1,4,5 In the US, 83.1% of pwCF are prescribed pancreatic enzyme replacement therapy (PERT) to manage EPI symptoms. 3,4 In cases of severe acinar damage, endocrine pancreatic insufficiency with resultant diabetes mellitus also can occur. 4 In addition to EPI, multiple other factors can cause or worsen fat malabsorption, including short gut with a history of meconium ileus (MI), hepatobiliary diseases, and intraluminal factors (bile salts deficiency, altered pH, intestinal microbiome dysbiosis, and small intestine bacterial overgrowth [SIBO]).…”

Section: Introductionmentioning

confidence: 99%

“…In the pancreas, the accumulation of hyperviscous mucus blocks the release of digestive enzymes with subsequent pancreatic acinar tissue destruction and exocrine pancreatic insufficiency (EPI) resulting in malabsorption of nutrients and malnutrition 1,4,5 . In the US, 83.1% of pwCF are prescribed pancreatic enzyme replacement therapy (PERT) to manage EPI symptoms 3,4 . In cases of severe acinar damage, endocrine pancreatic insufficiency with resultant diabetes mellitus also can occur 4 …”

Section: Introductionmentioning

confidence: 99%

“…In the US, 83.1% of pwCF are prescribed pancreatic enzyme replacement therapy (PERT) to manage EPI symptoms 3,4 . In cases of severe acinar damage, endocrine pancreatic insufficiency with resultant diabetes mellitus also can occur 4 …”

Section: Introductionmentioning

confidence: 99%

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Cystic fibrosis and fat malabsorption: Pathophysiology of the cystic fibrosis gastrointestinal tract and the impact of highly effective CFTR modulator therapy

McDonald,

Reid,

Pohl

et al. 2024

Nut in Clin Prac

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Cystic fibrosis (CF) is a progressive, genetic, multi‐organ disease affecting the respiratory, digestive, endocrine, and reproductive systems. CF can affect any aspect of the gastrointestinal (GI) tract, including the esophagus, stomach, small intestine, colon, pancreas, liver, and gall bladder. GI pathophysiology associated with CF results from CF membrane conductance regulator (CFTR) dysfunction. The majority of people with CF (pwCF) experience exocrine pancreatic insufficiency resulting in malabsorption of nutrients and malnutrition. Additionally, other factors can cause or worsen fat malabsorption, including the potential for short gut syndrome with a history of meconium ileus, hepatobiliary diseases, and disrupted intraluminal factors, such as inadequate bile salts, abnormal pH, intestinal microbiome changes, and small intestinal bacterial overgrowth. Signs and symptoms associated with fat malabsorption, such as abdominal pain, bloating, malodorous flatus, gastroesophageal reflux, nausea, anorexia, steatorrhea, constipation, and distal intestinal obstruction syndrome, are seen in pwCF despite the use of pancreatic enzyme replacement therapy. Given the association of poor nutrition status with lung function decline and increased mortality, aggressive nutrition support is essential in CF care to optimize growth in children and to achieve and maintain a healthy body mass index in adults. The introduction of highly effective CFTR modulator therapy and other advances in CF care have profoundly changed the course of CF management. However, GI symptoms in some pwCF may persist. The use of current knowledge of the pathophysiology of the CF GI tract as well as appropriate, individualized management of GI symptoms continue to be integral components of care for pwCF.

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Section: Pancreasmentioning

confidence: 99%

Section: Pathophysiology Of the Cf Gi Tractmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Cystic fibrosis and fat malabsorption: Pathophysiology of the cystic fibrosis gastrointestinal tract and the impact of highly effective CFTR modulator therapy

McDonald,

Reid,

Pohl

et al. 2024

Nut in Clin Prac

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Cystic fibrosis

Fajac

Burgel

2023

La Presse Médicale

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Preservation of β-cell Function in Pancreatic Insufficient Cystic Fibrosis With Highly Effective CFTR Modulator Therapy

Flatt,

Sheikh,

Peleckis

et al. 2023

The Journal of Clinical Endocrinology &Amp; Metabolism

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Context Elexacaftor/Tezacaftor/Ivacaftor (ETI; Trikafta™) enhances aberrant cystic fibrosis transmembrane conductance regulator (CFTR) function and may improve the insulin secretory defects associated with a deterioration in clinical outcomes in pancreatic insufficient cystic fibrosis (PI-CF). Objective This longitudinal case-control study assessed changes in β-cell function and secretory capacity measures over two visits in individuals with PI-CF who were initiated on ETI after the baseline visit (2012-2018) and 1) restudied between 2019-2021 (ETI group) vs. 2) those restudied between 2015-2018 and not yet treated with CFTR modulator therapy (controls). Methods Nine ETI participants (mean ± SD age 25 ± 5 years) and eight matched controls were followed up after median(IQR) 5(4-7) and 3(2-3) years, respectively (p < 0.01), with ETI initiation a median of 1 year before follow-up. Clinical outcomes, glucose-potentiated arginine (GPA) and mixed-meal tolerance test (MMTT) measures were assessed with comparisons of within and between group change by non-parametric testing. Results Glucose-potentiated insulin and C-peptide responses to GPA deteriorated in controls but not the ETI group, with C-peptide changes different between groups (p < 0.05). Deterioration in basal proinsulin secretory ratio was observed in controls but improved, as did the maximal arginine-induced proinsulin secretory ratio, in the ETI group (p < 0.05 for all comparisons). During MMTT, early insulin secretion improved as evidenced by more rapid insulin secretory rate kinetics. Conclusion ETI preserves β-cell function in CF through effects on glucose-dependent insulin secretion, proinsulin processing and meal-related insulin secretion. Further work should determine whether early intervention with ETI can prevent deterioration of glucose tolerance in PI-CF.

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Endocrine Complications of Cystic Fibrosis

Cited by 4 publications

References 121 publications

Cystic fibrosis and fat malabsorption: Pathophysiology of the cystic fibrosis gastrointestinal tract and the impact of highly effective CFTR modulator therapy

Cystic fibrosis and fat malabsorption: Pathophysiology of the cystic fibrosis gastrointestinal tract and the impact of highly effective CFTR modulator therapy

Cystic fibrosis

Preservation of β-cell Function in Pancreatic Insufficient Cystic Fibrosis With Highly Effective CFTR Modulator Therapy

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