Dataset Information

Delayed release pancrelipase for the treatment of pancreatic exocrine insufficiency associated with cystic fibrosis.

ABSTRACT: Pancreatic enzyme replacement therapy (PERT) is the only treatment for malabsorption in cystic fibrosis (CF) caused by pancreatic insufficiency (PI). PI occurs in approximately 85% of patients with CF. PERT overcomes some, but not all the signs and symptoms of malabsorption. Clinical parameters such as growth, abdominal pain, diarrhea and gassiness, commonly used to adjust PERT dosing, are shown not to be good indicators of their effectiveness. The FDA does not provide oversight of preparations of pancreatic enzymes consistent with the oversight it provides for all other drugs. The FDA intends to rectify this situation. Measures of the effectiveness of PERT are limited to the coefficient of fat absorption, a difficult and unpleasant exercise for patients.

SUBMITTER: Baker SS

PROVIDER: S-EPMC2621407 | biostudies-other | 2008 Oct

REPOSITORIES: biostudies-other

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Delayed release pancrelipase for the treatment of pancreatic exocrine insufficiency associated with cystic fibrosis.

Baker Susan S SS

Therapeutics and clinical risk management 20081001 5

Pancreatic enzyme replacement therapy (PERT) is the only treatment for malabsorption in cystic fibrosis (CF) caused by pancreatic insufficiency (PI). PI occurs in approximately 85% of patients with CF. PERT overcomes some, but not all the signs and symptoms of malabsorption. Clinical parameters such as growth, abdominal pain, diarrhea and gassiness, commonly used to adjust PERT dosing, are shown not to be good indicators of their effectiveness. The FDA does not provide oversight of preparations ...[more]

PMID: 19209287

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Project description:Exocrine pancreatic insufficiency (EPI) is a clinically defined syndrome based on the physician's assessment of a patient's maldigestion. However, current clinical definitions are inadequate in determining (1) the threshold of reduced pancreatic digestive enzyme secretion that determines "pancreatic insufficiency" in an individual patient; (2) the role of pancreatic function tests; (3) effects of differing metabolic needs, nutrition intake, and intestinal function/adaptation (4) when pancreatic enzyme replacement therapy is needed; and (5) how to monitor and titrate multiple therapies. Experts and key opinion leaders were invited to PancreasFest 2021 to discuss and help clarify mechanistic issues critical to defining EPI and to address misconceptions and barriers limiting advancements in patient care. Clinically EPI is defined as inadequate delivery of pancreatic digestive enzymes to meals to meet nutritional needs and is reversed with appropriate treatment. A new mechanistic definition of EPI was proposed that includes the disorders essence and character: (1) EPI is a disorder caused by failure of the pancreas to deliver a minimum/threshold level of specific pancreatic digestive enzymes to the intestine in concert with ingested nutrients, followed by enzymatic digestion of a series of individual snacks and meals over time to meet nutritional and metabolic needs, given (a) the specific macronutritional and micronutritional needs; (b) nutrient intake; (c) exocrine pancreatic function; and (d) intestinal anatomy, function, diseases, and adaptative capacity. (2) EPI is characterized by variable deficiencies in micronutrients and macronutrients, especially essential fats and fat-soluble vitamins, by gastrointestinal symptoms of nutrient maldigestion and by improvement or correction of nutritional state with lifestyle changes, disease treatment, optimized diet, dietary supplements, and/or administration of adequate pancreatic enzyme replacement therapy. EPI is complex and individualized and multidisciplinary approaches are needed to optimize therapy. Better pancreas function tests and biomarkers are needed to diagnose EPI and guide treatment.

Dataset Information

Delayed release pancrelipase for the treatment of pancreatic exocrine insufficiency associated with cystic fibrosis.

Publications

Delayed release pancrelipase for the treatment of pancreatic exocrine insufficiency associated with cystic fibrosis.

Similar Datasets

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