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.
RCT Entities:
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: Michael W Konstan; Drucy Borowitz; Nicole Mayer-Hamblett; Carlos Milla; Leslie Hendeles; Susan Murray; Richard A Kronmal; Susan Casey; Lynn M Rose; Wayne J Morgan; Bonnie W Ramsey Journal: Clin Investig (Lond) Date: 2013-08
Authors: Steven M Rowe; Drucy S Borowitz; Jane L Burns; John P Clancy; Scott H Donaldson; George Retsch-Bogart; Scott D Sagel; Bonnie W Ramsey Journal: Thorax Date: 2012-10 Impact factor: 9.139