title: Pancreatic enzyme replacement therapy dosing and nutritional outcomes in children with cystic fibrosis.
abstract: OBJECTIVE
  To utilize the Cystic Fibrosis Foundation Patient Registry to evaluate whether pancreatic enzyme dose is associated with better nutritional status as measured by average body mass index (BMI) percentile.
  
  
  METHODS
  A retrospective analysis of the Cystic Fibrosis Foundation Patient Registry from 2005-2008 was performed. The final analysis included 42 561 patient visits from 14 482 patients 2-20 years of age taking pancreatic enzyme replacement therapy from 179 programs. Cystic fibrosis care programs were assigned to quartiles based on adjusted mean patient BMI percentiles. Differences in median lipase dose between programs in the highest and lowest BMI quartiles were examined using a mixed effects model that adjusted for individual patient BMI, age, race, ethnicity, forced expiratory volume in 1 second percent, acid-blocker use, presence of Pseudomonas aeruginosa, nutritional supplement use, growth hormone use, and diagnosis of cystic fibrosis-related diabetes.
  
  
  RESULTS
  A significant difference in median enzyme dose existed between the highest and lowest BMI quartiles. Multivariable analysis demonstrated the effect persisted after adjustment for covariates. Highest quartile programs had a median enzyme dose of 1755 lipase units/kg/meal compared with 1628 lipase units/kg/meal for lowest quartile programs.
  
  
  CONCLUSIONS
  Patients attending US cystic fibrosis programs achieving highest nutritional outcomes, measured by mean BMI percentile, have higher enzyme dosing than those attending programs at lower performance levels. Further randomized clinical trials are necessary to determine the role of enzyme dose in improving nutritional outcomes.
authors.affiliation: Division of Pulmonary Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL. Electronic address: mark.haupt@abbvie.com.
authors.forename: Mark E
authors.initials: ME
authors.lastname: Haupt
authors.affiliation: Department of Preventive Medicine, Northwestern University, Chicago, IL.
authors.forename: Mary J
authors.initials: MJ
authors.lastname: Kwasny
authors.affiliation: Division of Pulmonary Medicine, Children's Hospital of Richmond, Virginia Commonwealth University, Richmond, VA.
authors.forename: Michael S
authors.initials: MS
authors.lastname: Schechter
authors.affiliation: Division of Pulmonary Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL.
authors.forename: Susanna A
authors.initials: SA
authors.lastname: McColley
mesh_terms: D000293:Adolescent; D015992:Body Mass Index; D002648:Child; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000150:complications*; D004334:Drug Administration Schedule; D056947:Enzyme Replacement Therapy / Q000379:methods*; D010188:Exocrine Pancreatic Insufficiency / Q000188:drug therapy* / Q000209:etiology; D005260:Female; D006801:Humans; D008049:Lipase / Q000627:therapeutic use*; D008297:Male; D015233:Models, Statistical; D015999:Multivariate Analysis; D009752:Nutritional Status; D012042:Registries; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D008049:Lipase