scholarly journals Study design considerations for evaluating the efficacy and safety of pancreatic enzyme replacement therapy in patients with cystic fibrosis

2013 ◽  
Vol 3 (8) ◽  
pp. 731-741 ◽  
Author(s):  
Michael W Konstan ◽  
Drucy Borowitz ◽  
Nicole Mayer-Hamblett ◽  
Carlos Milla ◽  
Leslie Hendeles ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Study Design ◽  
Pancreatic Enzyme ◽  
Efficacy And Safety ◽  
Enzyme Replacement ◽  
Design Considerations ◽  
Pancreatic Enzyme Replacement Therapy

scholarly journals The role of nutrition and pancreatic enzyme replacement therapy in children with cystic fibrosis

2021 ◽  
Vol 4 (2) ◽  
pp. 84-93
Author(s):  
Muzal Kadim ◽  
William Cheng
Keyword(s):  
Cystic Fibrosis ◽  
Nutritional Status ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pancreatic Insufficiency ◽  
Pancreatic Enzyme ◽  
Nutritional Intervention ◽  
Enzyme Replacement ◽  
Pancreatic Enzyme Replacement Therapy

Background Cystic fibrosis (CF) is an inherited genetic disorder with high mortality and morbidity. CF is strongly correlated with malnutrition due to higher energy losses, pancreatic insufficiency, chronic inflammation, higher resting energy expenditure, and feeding problems. Malnutrition in CF patients associated with worse survival. Thus, appropriate and prompt nutritional intervention should be addressed to reduced malnutrition in CF patients. Methods The literature search was performed on 9 August 2021 in four major databases such as MEDLINE, EBSCOhost, Cochrane Reviews, and Web of Sciences to find the role of nutrition and pancreatic enzyme replacement therapy in pediatrics population with cystic fibrosis. Recent findings In recent decades, early nutritional management and pancreatic enzyme replacement therapy (PERT) have been shown to improve CF patient’s outcomes. Nutrition should be given in higher calories compared to healthy individuals with close and regular nutritional status monitoring. High protein and fat diets are essential for CF patient’s overall survival. Adequate level of micronutrients should be ensured to avoid morbidity caused by micronutrients deficiency. Regular pancreatic insufficiency screening should be done annually in order to start PERT early.  Further research focusing on body composition, growth chart, protein intake, and PERT are needed to further improve the management of CF patient. Conclusion Nutritional intervention and PERT play an important role in prolonging CF patient survival. Both treatments should be initiated early with nutritional status close monitoring and tailored to each individual. Collaboration with parents and children is critical to warrant that CF patients followed the dietary advice.

scholarly journals Hospitalization rates among patients with cystic fibrosis using pancreatic enzyme replacement therapy

2020 ◽  
Vol 17 ◽  
pp. 147997311990061 ◽  
Author(s):  
Bruce C. Trapnell ◽  
Su Chen ◽  
Rupal Khurmi ◽  
Amit Bodhani ◽  
Mudra Kapoor ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Hospital Admissions ◽  
Pancreatic Enzyme ◽  
Admission Rate ◽  
Hospital Admission Rate ◽  
Enzyme Replacement ◽  
Pancreatic Enzyme Replacement Therapy ◽  
Annual Total

We investigated the relationship between self-reported adherence to pancreatic enzyme replacement therapy (PERT), nutritional status, and all-cause hospitalization in cystic fibrosis (CF) patients with a record of PERT use. Association of self-reported annual PERT use rate (adherence) with annual hospital admission rate (HAR) and annual total hospital nights (THNs) were analyzed for 5301 children (2000–2012) and 13,989 adults (2000–2013) from the CF Foundation Patient Registry. Multivariate linear regression was used to determine the association of HAR and THN with mean annual PERT use rate, cumulative PERT use rate, mean body mass index (BMI) (adult) or BMI percentile (pediatric), age, and sex. The median annual PERT use rate was 87% in children and 80% in adults. Statistically, higher annual PERT use, longer cumulative PERT, and higher BMI percentile (children) or BMI (adults) were significantly ( p < 0.0001) associated with lower annual HAR and fewer annual THN in children and adults. Female sex was associated with higher annual HAR and more annual THN in children and adults ( p < 0.05). Results indicate self-reported adherence to PERT, increased BMI, and male sex were associated with fewer hospital admissions and annual hospital nights in CF patients.

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