Literature DB >> 25132954

Study design considerations for evaluating the efficacy and safety of pancreatic enzyme replacement therapy in patients with cystic fibrosis.

Michael W Konstan1, Drucy Borowitz2, Nicole Mayer-Hamblett3, Carlos Milla4, Leslie Hendeles5, Susan Murray6, Richard A Kronmal3, Susan Casey7, Lynn M Rose3, Wayne J Morgan8, Bonnie W Ramsey3.   

Abstract

In 2006, the US FDA issued a 'Guidance for Industry' regarding submission of New Drug Applications for pancreatic enzyme replacement therapy (PERT) products. Five oral delayed-release PERT products have been approved by the FDA, and several others are under development and/ or evaluation for New Drug Application submission. We present in this paper recommendations of the Cystic Fibrosis Foundation's Cystic Fibrosis (CF) Therapeutics Development Network and Data Safety Monitoring Board regarding study design considerations for evaluating PERT products in patients with CF. Careful attention to study design and accuracy of the outcome measures has confirmed our understanding of the efficacy and safety of PERT for the treatment of exocrine pancreatic insufficiency of CF.

Entities:  

Keywords:  coefficient of fat absorption; cystic fibrosis; pancreatic enzyme replacement therapy; pancreatic insufficiency; study design

Year:  2013        PMID： 25132954      PMCID： PMC4131768          DOI： 10.4155/cli.13.63

Source DB:  PubMed          Journal:  Clin Investig (Lond)        ISSN： 2041-6792


  42 in total

1.  Comparison of steatocrit and fat absorption in persons with cystic fibrosis.

Authors:  Mary H Wagner; Ellen K Bowser; James M Sherman; Mary Pat Francisco; Douglas Theriaque; Donald A Novak
Journal:  J Pediatr Gastroenterol Nutr       Date:  2002-08       Impact factor: 2.839

2.  13Carbon mixed triglyceride breath test and pancreatic enzyme supplementation in cystic fibrosis.

Authors:  S Amarri; M Harding; W A Coward; T J Evans; L T Weaver
Journal:  Arch Dis Child       Date:  1997-04       Impact factor: 3.791

3.  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.

Authors:  R C Stern; J D Eisenberg; J S Wagener; R Ahrens; M Rock; G doPico; D M Orenstein
Journal:  Am J Gastroenterol       Date:  2000-08       Impact factor: 10.864

4.  High-dose pancreatic-enzyme supplements and fibrosing colonopathy in children with cystic fibrosis.

Authors:  S C FitzSimmons; G A Burkhart; D Borowitz; R J Grand; T Hammerstrom; P R Durie; J D Lloyd-Still; A B Lowenfels
Journal:  N Engl J Med       Date:  1997-05-01       Impact factor: 91.245

5.  Uric acid metabolism in children.

Authors:  L A Baldree; F B Stapleton
Journal:  Pediatr Clin North Am       Date:  1990-04       Impact factor: 3.278

6.  Biokinetics in humans of RRR-alpha-tocopherol: the free phenol, acetate ester, and succinate ester forms of vitamin E.

Authors:  K H Cheeseman; A E Holley; F J Kelly; M Wasil; L Hughes; G Burton
Journal:  Free Radic Biol Med       Date:  1995-11       Impact factor: 7.376

7.  Efficacy and safety of Creon 24,000 in subjects with exocrine pancreatic insufficiency due to cystic fibrosis.

Authors:  Bruce C Trapnell; Karen Maguiness; Gavin R Graff; David Boyd; Katrin Beckmann; Steven Caras
Journal:  J Cyst Fibros       Date:  2009-10-07       Impact factor: 5.482

8.  Cystic Fibrosis Foundation evidence-based guidelines for management of infants with cystic fibrosis.

Authors:  Drucy Borowitz; Karen A Robinson; Margaret Rosenfeld; Stephanie D Davis; Kathryn A Sabadosa; Stephanie L Spear; Suzanne H Michel; Richard B Parad; Terry B White; Philip M Farrell; Bruce C Marshall; Frank J Accurso
Journal:  J Pediatr       Date:  2009-12       Impact factor: 4.406

9.  Strictures of ascending colon in cystic fibrosis and high-strength pancreatic enzymes.

Authors:  R L Smyth; D van Velzen; A R Smyth; D A Lloyd; D P Heaf
Journal:  Lancet       Date:  1994-01-08       Impact factor: 79.321

Review 10.  Evidence-based practice recommendations for nutrition-related management of children and adults with cystic fibrosis and pancreatic insufficiency: results of a systematic review.

Authors:  Virginia A Stallings; Lori J Stark; Karen A Robinson; Andrew P Feranchak; Hebe Quinton
Journal:  J Am Diet Assoc       Date:  2008-05
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  3 in total

1.  Efficacy and safety of a unique enteric-coated bicarbonate-buffered pancreatic enzyme replacement therapy in children and adults with cystic fibrosis.

Authors:  Michael W Konstan; Frank J Accurso; Samya Z Nasr; Richard C Ahrens; Gavin R Graff
Journal:  Clin Investig (Lond)       Date:  2013-08-01

2.  Coefficient of Fat Absorption to Measure the Efficacy of Pancreatic Enzyme Replacement Therapy in People With Cystic Fibrosis: Gold Standard or Coal Standard?

Authors:  Drucy Borowitz; Nell Aronoff; Linda C Cummings; Asim Maqbool; Andrew E Mulberg
Journal:  Pancreas       Date:  2022-06-11       Impact factor: 3.243

Review 3.  Timing of pancreatic enzyme replacement therapy (PERT) in cystic fibrosis.

Authors:  Christabella Ng; Giles Major; Alan R Smyth
Journal:  Cochrane Database Syst Rev       Date:  2021-08-02
  3 in total

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