title: Efficacy and safety of PANCREAZE® for treatment of exocrine pancreatic insufficiency due to cystic fibrosis.
abstract: BACKGROUND
  Pancreatic enzyme replacement therapy (PERT) is critical for correction of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF).
  
  
  METHODS
  This was a randomized, placebo-controlled PERT withdrawal study evaluating the efficacy and safety of PANCREAZE® (pancrelipase) in CF patients with EPI. Participants (n=49) entered an open-label, ≤ 14 day run-in phase, maintained a high-fat diet (100 ± 15 g/day), and received PANCREAZE® (10.5 or 21). Participants with a coefficient of fat absorption (CFA)≥ 80% (n=40) were then randomized (1:1) to receive either PANCREAZE® or placebo during a double-blind, ≤ 7 day withdrawal phase.
  
  
  RESULTS
  PANCREAZE® improved fat absorption as shown by significantly lower mean ± SD change in CFA between open-label and double-blind phases for PANCREAZE® (-1.5 ± 5.88%; p<0.001) compared to placebo (-34.1 ± 23.03%). Protein absorption was similarly improved. No unexpected adverse events were reported.
  
  
  CONCLUSIONS
  This study demonstrated PANCREAZE® was effective in treating EPI due to CF and was safe and well tolerated.
authors.affiliation: Division of Pulmonary Medicine and Biology, Cincinnati Children's Hospital Medical Center, OH, USA. Bruce.Trapnell@cchmc.org
authors.forename: Bruce C
authors.initials: BC
authors.lastname: Trapnell
authors.forename: Steven D
authors.initials: SD
authors.lastname: Strausbaugh
authors.forename: Marlyn S
authors.initials: MS
authors.lastname: Woo
authors.forename: Shin-Yir
authors.initials: SY
authors.lastname: Tong
authors.forename: Steven A
authors.initials: SA
authors.lastname: Silber
authors.forename: Andrew E
authors.initials: AE
authors.lastname: Mulberg
authors.forename: Gerhard
authors.initials: G
authors.lastname: Leitz
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D003550:Cystic Fibrosis / Q000150:complications*; D010188:Exocrine Pancreatic Insufficiency / Q000188:drug therapy*; D005260:Female; D005765:Gastrointestinal Agents / Q000008:administration & dosage* / Q000009:adverse effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D046790:Pancreas, Exocrine / Q000187:drug effects*; D020799:Pancrelipase / Q000008:administration & dosage* / Q000009:adverse effects; D010919:Placebos; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005765:Gastrointestinal Agents; D010919:Placebos; D020799:Pancrelipase