Pancreatic complications in children with cystic fibrosis
1Pediatric Gastroenterology, Hepatology, and Nutrition, Stanford University, Stanford, California, USA.
Insights
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are improving exocrine pancreatic function in children, challenging previous notions of irreversibility. Research also reveals complex exocrine-endocrine pancreas interactions impacting diabetes risk.
Area of Science:
- Pediatric Gastroenterology
- Cystic Fibrosis Research
- Pancreatic Pathophysiology
Background:
- Pancreatic complications are a major cause of morbidity in children with cystic fibrosis (CF).
- Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) dysfunction significantly impacts exocrine and endocrine pancreatic function from early childhood.
- Understanding these pancreatic effects is crucial for managing CF-related complications.
Purpose of the Study:
- To review recent research on exocrine pancreatic function in children with CF, focusing on CFTR modulator therapies.
- To discuss the impact of these therapies on pancreatitis and exocrine pancreatic insufficiency (EPI).
- To explore new insights into exocrine-endocrine interactions within the pancreas in CF.
Main Methods:
- Literature review of recent studies on CFTR modulators and pancreatic function in pediatric CF.
- Analysis of research on pancreatitis and EPI in children undergoing CFTR modulator therapy.
- Examination of emerging research on exocrine-endocrine pancreatic crosstalk in CF.
Main Results:
- CFTR modulator therapies, such as ivacaftor, have demonstrated improvements in exocrine pancreatic function, challenging the concept of irreversible EPI.
- Pancreatic function improvements have led to varied effects on pancreatitis episodes.
- Advances in imaging offer complementary diagnostic tools for exocrine pancreatic function.
Conclusions:
- CFTR modulators are transforming the management of pancreatic complications in pediatric CF.
- Further research into pancreatic gene therapy and exocrine-endocrine interactions holds promise for novel therapeutic strategies.
- These advancements aim to reduce morbidity and mortality associated with pancreatitis, EPI, and diabetes in CF.
Purpose Of Review:
The pancreas is highly affected in cystic fibrosis, with complications occurring early in childhood. This review highlights recent research in exocrine pancreatic function in the era of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies and discusses how these are affecting pancreatitis and exocrine pancreatic insufficiency (EPI) in children. Additionally, new research into exocrine--endocrine interactions sheds light on how CFTR dysfunction in ductal cells may affect beta cells.
Recent Findings:
Ivacaftor has disproved the hypothesis that EPI in children with cystic fibrosis is irreversible. Improvements in pancreatic function have increased pancreatitis episodes in some children and reduced them in others. Imaging advances are providing complementary methods for exocrine pancreatic function testing. New research into the interplay between the exocrine and endocrine components of the pancreas are elucidating the intertwined and complex relationship between the exocrine and endocrine pancreas.
Summary:
Pancreatic complications contribute to the morbidity and mortality of children with cystic fibrosis. Increasing use of highly effective CFTR modulators will not only abrogate these but will also advance our understanding of pancreatic pathophysiology in cystic fibrosis. New frontiers into pancreatic gene therapy and exocrine--endocrine research will help provide new therapeutic opportunities for pancreatitis, EPI, and diabetes in cystic fibrosis.
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