title: 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.
abstract: OBJECTIVE
  The safety and efficacy of Minimicrospheres, which are enteric-coated, delayed-release pancrelipase capsules, on fat absorption in pediatric/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 insufficiency and steatorrhea were stabilized on a high-fat diet and administered pancrelipase. Seventy-four patients with >80% coefficient of fat absorption received placebo or pancrelipase in the double-blind phase. Fat intake and excretion, stool frequency and consistency, and clinical global improvement were recorded.
  
  
  RESULTS
  Average daily fat intake was comparable between treatment groups within each age group (adults vs pediatric/adolescent), but placebo patients had a significant (p < 0.001) mean decrease in coefficient of fat absorption (adult, 36.9 percentage points; pediatric/adolescent, 34.9 percentage points) from open-label to double-blind treatment compared to pancrelipase patients (adult, 2 percentage points; pediatric/adolescent, 3.25 percentage points); this difference was caused by a greater (p < or = 0.001) increase in mean fecal fat excretion (grams per day) in the placebo groups compared to pancrelipase groups (adult: 61.9 vs 2.3; pediatric/adolescent: 45.4 vs 4.1). Change in mean stool frequency from open-label to double-blind phases was significantly different (p < or = 0.002) between treatment groups, with increases in placebo groups and no difference (adult) or decrease (pediatric/adolescent) in pancrelipase groups. Pancrelipase patients' stool consistency remained about the same from open-label to double-blind. Placebo patients' stool consistency decreased (became softer) from open-label pancrelipase to double-blind placebo. Clinical global improvement data showed that > or =83% of pancrelipase patients improved or remained unchanged.
  
  
  CONCLUSIONS
  Enteric-coated, delayed-release (Minimicrospheres) pancrelipase capsules are an effective treatment for steatorrhea associated with pancreatic insufficiency in patients with cystic fibrosis.
authors.affiliation: Rainbow Babies & Children's Hospital, Cleveland, Ohio 44106, USA.
authors.forename: R C
authors.initials: RC
authors.lastname: Stern
authors.forename: J D
authors.initials: JD
authors.lastname: Eisenberg
authors.forename: J S
authors.initials: JS
authors.lastname: Wagener
authors.forename: R
authors.initials: R
authors.lastname: Ahrens
authors.forename: M
authors.initials: M
authors.lastname: Rock
authors.forename: G
authors.initials: G
authors.lastname: doPico
authors.forename: D M
authors.initials: DM
authors.lastname: Orenstein
mesh_terms: D000328:Adult; D002446:Celiac Disease / Q000188:drug therapy* / Q000209:etiology*; D003550:Cystic Fibrosis / Q000150:complications*; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D010188:Exocrine Pancreatic Insufficiency / Q000209:etiology*; D005260:Female; D005765:Gastrointestinal Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D020799:Pancrelipase / Q000009:adverse effects* / Q000627:therapeutic use*; D010919:Placebos / Q000009:adverse effects / Q000627:therapeutic use; D013997:Time Factors
chemical_list: D005765:Gastrointestinal Agents; D010919:Placebos; D020799:Pancrelipase