Community Perspective on Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis. 2022

Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
From the University at Buffalo Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY.

People with cystic fibrosis (CF) and exocrine pancreatic insufficiency must take pancreatic enzyme replacement therapy (PERT) to prevent malnutrition and gastrointestinal (GI) symptoms. Finding better ways to manage GI complaints is a high priority for the CF community. We fielded a survey to assess the perspective of people affected by CF regarding symptoms attributed to and challenges associated with current PERT, to identify factors that affect participation in PERT studies, and to understand attitudes toward an outcome measure that could be an alternative to the coefficient of fat absorption test. Persistent GI symptoms are commonly ascribed to PERT. Minimizing time commitment and maximizing patient safety were factors affecting participation in research. We demonstrate 4 generalizable ways to incorporate patient experience early in the research process to aid in development of new medications and help improve study enrollment.

UI MeSH Term Description Entries
D010179 Pancreas A nodular organ in the ABDOMEN that contains a mixture of ENDOCRINE GLANDS and EXOCRINE GLANDS. The small endocrine portion consists of the ISLETS OF LANGERHANS secreting a number of hormones into the blood stream. The large exocrine portion (EXOCRINE PANCREAS) is a compound acinar gland that secretes several digestive enzymes into the pancreatic ductal system that empties into the DUODENUM.
D010188 Exocrine Pancreatic Insufficiency A malabsorption condition resulting from greater than 10% reduction in the secretion of pancreatic digestive enzymes (LIPASE; PROTEASES; and AMYLASE) by the EXOCRINE PANCREAS into the DUODENUM. This condition is often associated with CYSTIC FIBROSIS and with chronic PANCREATITIS. Pancreatic Insufficiency,Exocrine Pancreatic Insufficiencies,Insufficiencies, Exocrine Pancreatic,Insufficiencies, Pancreatic,Insufficiency, Exocrine Pancreatic,Insufficiency, Pancreatic,Pancreatic Insufficiencies,Pancreatic Insufficiencies, Exocrine,Pancreatic Insufficiency, Exocrine
D003550 Cystic Fibrosis An autosomal recessive genetic disease of the EXOCRINE GLANDS. It is caused by mutations in the gene encoding the CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR expressed in several organs including the LUNG, the PANCREAS, the BILIARY SYSTEM, and the SWEAT GLANDS. Cystic fibrosis is characterized by epithelial secretory dysfunction associated with ductal obstruction resulting in AIRWAY OBSTRUCTION; chronic RESPIRATORY INFECTIONS; PANCREATIC INSUFFICIENCY; maldigestion; salt depletion; and HEAT PROSTRATION. Mucoviscidosis,Cystic Fibrosis of Pancreas,Fibrocystic Disease of Pancreas,Pancreatic Cystic Fibrosis,Pulmonary Cystic Fibrosis,Cystic Fibrosis, Pancreatic,Cystic Fibrosis, Pulmonary,Fibrosis, Cystic,Pancreas Fibrocystic Disease,Pancreas Fibrocystic Diseases
D005767 Gastrointestinal Diseases Diseases in any segment of the GASTROINTESTINAL TRACT from ESOPHAGUS to RECTUM. Cholera Infantum,Gastrointestinal Disorders,Functional Gastrointestinal Disorders,Gastrointestinal Disorders, Functional,Disease, Gastrointestinal,Diseases, Gastrointestinal,Functional Gastrointestinal Disorder,Gastrointestinal Disease,Gastrointestinal Disorder,Gastrointestinal Disorder, Functional
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D044342 Malnutrition An imbalanced nutritional status resulting from insufficient intake of nutrients to meet normal physiological requirement. Malnourishment,Nutritional Deficiency,Undernutrition,Malnourishments,Nutritional Deficiencies
D056947 Enzyme Replacement Therapy Therapeutic replacement or supplementation of defective or missing enzymes to alleviate the effects of enzyme deficiency (e.g., GLUCOSYLCERAMIDASE replacement for GAUCHER DISEASE). Enzyme Replacement Therapies,Replacement Therapies, Enzyme,Replacement Therapy, Enzyme,Therapies, Enzyme Replacement,Therapy, Enzyme Replacement

Related Publications

Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
March 2022, Nutrients,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
January 2009, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
November 2016, The Cochrane database of systematic reviews,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
October 2014, The Cochrane database of systematic reviews,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
August 2020, The Cochrane database of systematic reviews,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
August 2021, The Cochrane database of systematic reviews,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
August 2006, Journal of the American Dietetic Association,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
March 2015, Paediatric respiratory reviews,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
December 2008, Expert review of respiratory medicine,
Drucy Borowitz, and Joan Finnegan Brooks, and Enid Aliaj, and Danielle Gallotto
August 2013, Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition,
Copied contents to your clipboard!