Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency.

Abstract

OBJECTIVE:

The standard measure of pancreatic enzyme replacement therapy (PERT) efficacy in treating exocrine pancreatic insufficiency (EPI) is the coefficient of fat absorption (CFA). CFA measurement involves 72-hour stool collection, which presents a logistical challenge because, although the test may be performed on an outpatient basis in clinical practice, hospitalization is needed if assurance of complete collection and 100% compliance is required, for example, in controlled situations such as clinical trials. Our aim was to investigate sparse stool sample collection as an alternative to complete 72-hour collection for measurement of stool fat in subjects with EPI.

SUBJECTS AND METHODS:

Prospective data analysis from a previously published, double-blind, randomized, placebo-controlled, 2-period crossover trial in subjects ages 7 to 11 years with EPI caused by cystic fibrosis. Percentage fat (PF) data from sparse stool samples were compared with 72-hour CFA values as a dichotomous variable (<80%, ≥80%), with evaluation of sensitivity, specificity, and positive predictive value. Area under the curve values were obtained from receiver operating characteristic plots of sensitivity versus 1-specificity.

RESULTS:

Twelve subjects provided samples for this analysis. Multiple-sample PF values ≤30% were greatly predictive for CFA values ≥80%, as shown by positive predictive value, sensitivity, and specificity values ≥0.89, with high accuracy (AUCs ≥0.93).

CONCLUSIONS:

Sparse stool sampling for PF analysis appears to be a valid, practical alternative to 72-hour CFA determination and has potential as a screening tool in clinical practice to identify both suboptimal dosing in subjects with EPI receiving PERT and substantial fat malabsorption in subjects not receiving PERT.

• Authors: Steve Caras, David Boyd, Lisa Zipfel, Suntje Sander-Struckmeier
• Journal: Journal of pediatric gastroenterology and nutrition (J Pediatr Gastroenterol Nutr) Vol. 53 Issue 6 Pg. 634-40 (Dec 2011) ISSN: 1536-4801 [Electronic] United States
• PMID: 21681115 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Fats
• Pancrelipase

Topics

• Child
• Cross-Over Studies
• Cystic Fibrosis (complications, physiopathology)
• Double-Blind Method
• Enzyme Replacement Therapy (methods)
• Exocrine Pancreatic Insufficiency (diagnosis, etiology)
• Fats (analysis)
• Feces
• Female
• Humans
• Intestinal Absorption (drug effects)
• Male
• Pancreas (drug effects, physiopathology)
• Pancrelipase (administration & dosage)
• Predictive Value of Tests
• Prospective Studies
• Randomized Controlled Trials as Topic
• Sensitivity and Specificity