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Treatment of infants and toddlers with cystic fibrosis-related pancreatic insufficiency and fat malabsorption with pancrelipase MT

Els Van de Vijver, Kristien Desager, Andrew E Mulberg, Sophie Staelens, Henk Jan Verkade, Frank AJA Bodewes, Anne Malfroot, Bruno Hauser, Maarten Sinaasappel and Stephanie Van Biervliet UGent, et al. (2011) JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION. 53(1). p.61-64
abstract
Background: Pancreatic enzyme replacement therapy (PERT) improves nutritional status and growth in patients with cystic fibrosis (CF) with pancreatic insufficiency (PI). The current recommendation for infants and young children, who are not able to swallow the whole capsule, is to open the capsule and mix the beads in a spoon with some applesauce; however, the efficacy and safety data of this approach are currently lacking. The aim of this study was to assess the efficacy, palatability (ease of swallowing), and safety of 4 dose levels of pancrelipase microtablets (Pancrease MT) in infants and young children with CF-related PI. Patients and Methods: This study was a phase II randomized, investigator-blinded, parallel-group pilot study in DNA-proven infants with CF and PI. The study design included a run-in period (days 1-5) and an experimental period (days 6-11). Pancrelipase microtablets (2-mm, enteric coated) were provided orally. Sixteen subjects, 6 to 30 months of age, were provided 500 U lipase/kg/meal for 5 days (baseline period). Subsequently, subjects were randomly assigned to 1 of 4 treatment groups (each n = 4), receiving 500, 1000, 1500, or 2000 U (Ph. EUR) of lipase/kg/meal, respectively, for 5 days (experimental period). The primary endpoint was medication efficacy assessed by the 72-hour fecal fat excretion, expressed as coefficient of fecal fat absorption (CFA), and (13)C mixed triglyceride breath test. Secondary endpoints were safety and palatability. Results: Overall compliance, defined as used study medication, was 89% to 99% for the entire study. None of the 4 dose regimens significantly influenced the CFA, relative to the baseline period (median range 83%-93%). During the run-in period the median cumulative % (13)C was 11 (range -8 to 59). After randomization the median cumulative % (13)C was 18 (range 14-23) in the 500-U, 14 (range -1 to 17) in the 1000-U, 10 (range 10-27) in the 1500-U, and 3 (range 1-49) in the 2000-U groups. Palatability was scored fair to good by the parents in each of the treatment groups. Gastrointestinal symptoms were reported in some patients, including common adverse events reported in clinical trials involving pancreatic enzyme therapy. No serious or other adverse events were reported. Conclusion: Treatment with Pancrease MT at a dosage of 500 U lipase/kg/meal resulted in a CFA of approximately 89% in pediatric subjects ages 6 to 30 months with PI resulting from CF. Pancrease MT doses were well tolerated and mean palatability was scored as fair to good. Present results do not indicate that a dosage higher than 500 U (Ph. EUR) lipase/kg/meal increases the coefficient of fat absorption in a cohort of infants 6 to 30 months of age.
Please use this url to cite or link to this publication:
author
organization
year
type
journalArticle (original)
publication status
published
subject
keyword
infants, fat malabsorption, cystic fibrosis, 13)C mixed triglyceride breath test, pancreatic exocrine insufficiency, pancrelipase therapy, TRIGLYCERIDE BREATH TEST, ENZYME REPLACEMENT THERAPY, SUPPLEMENTS, COLONOPATHY, CHILDREN
journal title
JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION
J. Pediatr. Gastroenterol. Nutr.
volume
53
issue
1
pages
61 - 64
Web of Science type
Article
Web of Science id
000291925500009
JCR category
PEDIATRICS
JCR impact factor
2.298 (2011)
JCR rank
27/113 (2011)
JCR quartile
1 (2011)
ISSN
0277-2116
DOI
10.1097/MPG.0b013e31820e208e
language
English
UGent publication?
yes
classification
A1
copyright statement
I have transferred the copyright for this publication to the publisher
id
1939569
handle
http://hdl.handle.net/1854/LU-1939569
date created
2011-11-07 17:21:26
date last changed
2011-11-16 16:11:48
@article{1939569,
  abstract     = {Background: Pancreatic enzyme replacement therapy (PERT) improves nutritional status and growth in patients with cystic fibrosis (CF) with pancreatic insufficiency (PI). The current recommendation for infants and young children, who are not able to swallow the whole capsule, is to open the capsule and mix the beads in a spoon with some applesauce; however, the efficacy and safety data of this approach are currently lacking. The aim of this study was to assess the efficacy, palatability (ease of swallowing), and safety of 4 dose levels of pancrelipase microtablets (Pancrease MT) in infants and young children with CF-related PI. 
Patients and Methods: This study was a phase II randomized, investigator-blinded, parallel-group pilot study in DNA-proven infants with CF and PI. The study design included a run-in period (days 1-5) and an experimental period (days 6-11). Pancrelipase microtablets (2-mm, enteric coated) were provided orally. Sixteen subjects, 6 to 30 months of age, were provided 500 U lipase/kg/meal for 5 days (baseline period). Subsequently, subjects were randomly assigned to 1 of 4 treatment groups (each n = 4), receiving 500, 1000, 1500, or 2000 U (Ph. EUR) of lipase/kg/meal, respectively, for 5 days (experimental period). The primary endpoint was medication efficacy assessed by the 72-hour fecal fat excretion, expressed as coefficient of fecal fat absorption (CFA), and (13)C mixed triglyceride breath test. Secondary endpoints were safety and palatability. 
Results: Overall compliance, defined as used study medication, was 89\% to 99\% for the entire study. None of the 4 dose regimens significantly influenced the CFA, relative to the baseline period (median range 83\%-93\%). During the run-in period the median cumulative \% (13)C was 11 (range -8 to 59). After randomization the median cumulative \% (13)C was 18 (range 14-23) in the 500-U, 14 (range -1 to 17) in the 1000-U, 10 (range 10-27) in the 1500-U, and 3 (range 1-49) in the 2000-U groups. Palatability was scored fair to good by the parents in each of the treatment groups. Gastrointestinal symptoms were reported in some patients, including common adverse events reported in clinical trials involving pancreatic enzyme therapy. No serious or other adverse events were reported. 
Conclusion: Treatment with Pancrease MT at a dosage of 500 U lipase/kg/meal resulted in a CFA of approximately 89\% in pediatric subjects ages 6 to 30 months with PI resulting from CF. Pancrease MT doses were well tolerated and mean palatability was scored as fair to good. Present results do not indicate that a dosage higher than 500 U (Ph. EUR) lipase/kg/meal increases the coefficient of fat absorption in a cohort of infants 6 to 30 months of age.},
  author       = {Van de Vijver, Els and Desager , Kristien and Mulberg, Andrew E and Staelens, Sophie and Verkade, Henk Jan and Bodewes, Frank AJA and Malfroot, Anne and Hauser, Bruno and Sinaasappel, Maarten and Van Biervliet, Stephanie and Behm, Martin and Pelckmans, Paul and Callens, Dirk and Veereman-Wauters , Gigi},
  issn         = {0277-2116},
  journal      = {JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION},
  keyword      = {infants,fat malabsorption,cystic fibrosis,13)C mixed triglyceride breath test,pancreatic exocrine insufficiency,pancrelipase therapy,TRIGLYCERIDE BREATH TEST,ENZYME REPLACEMENT THERAPY,SUPPLEMENTS,COLONOPATHY,CHILDREN},
  language     = {eng},
  number       = {1},
  pages        = {61--64},
  title        = {Treatment of infants and toddlers with cystic fibrosis-related pancreatic insufficiency and fat malabsorption with pancrelipase MT},
  url          = {http://dx.doi.org/10.1097/MPG.0b013e31820e208e},
  volume       = {53},
  year         = {2011},
}

Chicago
Van de Vijver, Els, Kristien Desager , Andrew E Mulberg, Sophie Staelens, Henk Jan Verkade, Frank AJA Bodewes, Anne Malfroot, et al. 2011. “Treatment of Infants and Toddlers with Cystic Fibrosis-related Pancreatic Insufficiency and Fat Malabsorption with Pancrelipase MT.” Journal of Pediatric Gastroenterology and Nutrition 53 (1): 61–64.
APA
Van de Vijver, E., Desager , K., Mulberg, A. E., Staelens, S., Verkade, H. J., Bodewes, F. A., Malfroot, A., et al. (2011). Treatment of infants and toddlers with cystic fibrosis-related pancreatic insufficiency and fat malabsorption with pancrelipase MT. JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION, 53(1), 61–64.
Vancouver
1.
Van de Vijver E, Desager K, Mulberg AE, Staelens S, Verkade HJ, Bodewes FA, et al. Treatment of infants and toddlers with cystic fibrosis-related pancreatic insufficiency and fat malabsorption with pancrelipase MT. JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION. 2011;53(1):61–4.
MLA
Van de Vijver, Els, Kristien Desager , Andrew E Mulberg, et al. “Treatment of Infants and Toddlers with Cystic Fibrosis-related Pancreatic Insufficiency and Fat Malabsorption with Pancrelipase MT.” JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION 53.1 (2011): 61–64. Print.