A comparison of the efficacy and tolerance of pancrelipase and placebo in the treatment of steatorrhea in cystic fibrosis patients with clinical exocrine pancreatic insufficiency

OBJECTIVE: The safety and efficacy of Minimicrospheres, which are enteric-c oated, delayed-release pancrelipase capsules, on fat absorption in pediatri c/adolescent and adult cystic fibrosis (CF) patients was assessed. Exocrine pancreatic insufficiency, common in CF patients, causes steatorrhea due to insufficient release of pancreatic enzymes. METHODS: In the open-label phase, 97 CF patients with pancreatic insufficie ncy and steatorrhea were stabilized on a high-fat diet and administered pan crelipase. Seventy-four patients with >80% coefficient of fat absorption re ceived placebo or pancrelipase in the double-blind phase. Fat intake and ex cretion, stool frequency and consistency, and clinical global improvement w ere recorded. RESULTS: Average daily fat intake was comparable between treatment groups w ithin each age group (adults vs pediatric/adolescent), but placebo patients had a significant (p < 0.001) mean decrease in coefficient of fat absorpti on (adult, 36.9 percentage points; pediatric/adolescent, 34.9 percentage po ints) from open-label to double-blind treatment compared to pancrelipase pa tients (adult, 2 percentage points; pediatric/adolescent, 3.25 percentage p oints); this difference was caused by a greater (p less than or equal to 0. 001) increase in mean fecal fat excretion (grams per day) in the placebo gr oups compared to pancrelipase groups (adult: 61.9 vs 2.3; pediatric/adolesc ent: 45.4 vs 4.1). Change in mean stool frequency from open-label to double -blind phases was significantly different (p less than or equal to 0.002) b etween treatment groups, with increases in placebo groups and no difference (adult) or decrease (pediatric/adolescent) in pancrelipase groups. Pancrel ipase patients' stool consistency remained about the same from open-label t o double-blind. Placebo patients' stool consistency decreased (became softe r) from open-label pancrelipase to double-blind placebo. Clinical global im provement data showed that greater than or equal to 83% of pancrelipase pat ients improved or remained unchanged. CONCLUSIONS: Enteric-coated, delayed-release (Minimicrospheres) pancrelipas e capsules are an effective treatment for steatorrhea associated with pancr eatic insufficiency in patients with cystic (C) 2000 by Am. Cell, of Gastro enterology.