title: Protein and calorie intakes in adult and pediatric subjects with urea cycle disorders participating in clinical trials of glycerol phenylbutyrate.
abstract: BACKGROUND
  Little prospectively collected data are available comparing the dietary intake of urea cycle disorder (UCD) patients to UCD treatment guidelines or to healthy individuals.
  
  
  OBJECTIVE
  To examine the protein and calorie intakes of UCD subjects who participated in clinical trials of glycerol phenylbutyrate (GPB) and compare these data to published UCD dietary guidelines and nutritional surveys.
  
  
  METHODS
  Dietary data were recorded for 45 adult and 49 pediatric UCD subjects in metabolic control during participation in clinical trials of GPB. Protein and calorie intakes were compared to UCD treatment guidelines, average nutrient intakes of a healthy US population based on the National Health and Nutrition Examination Survey (NHANES) and Recommended Daily Allowances (RDA).
  
  
  RESULTS
  In adults, mean protein intake was higher than UCD recommendations but lower than RDA and NHANES values, while calorie intake was lower than UCD recommendations, RDA and NHANES. In pediatric subjects, prescribed protein intake was higher than UCD guidelines, similar to RDA, and lower than NHANES data for all age groups, while calorie intake was at the lower end of the recommended UCD range and close to RDA and NHANES data. In pediatric subjects height, weight, and body mass index (BMI) Z-scores were within normal range (- 2 to 2).
  
  
  CONCLUSIONS
  Pediatric patients treated with phenylbutyrate derivatives exhibited normal height and weight. Protein and calorie intakes in adult and pediatric UCD subjects differed from UCD dietary guidelines, suggesting that these guidelines may need to be reconsidered.
authors.affiliation: Miller Children's Hospital/Long Beach Medical Center, United States.
authors.forename: Debra
authors.initials: D
authors.lastname: Hook
authors.affiliation: Mount Sinai School of Medicine, Department of Genetics and Genomic Sciences, Department of Pediatrics, United States.
authors.forename: George A
authors.initials: GA
authors.lastname: Diaz
authors.affiliation: Baylor College of Medicine, United States.
authors.forename: Brendan
authors.initials: B
authors.lastname: Lee
authors.affiliation: Miller Children's Hospital/Long Beach Medical Center, United States.
authors.forename: James
authors.initials: J
authors.lastname: Bartley
authors.affiliation: The University of Utah, United States.
authors.forename: Nicola
authors.initials: N
authors.lastname: Longo
authors.affiliation: Stanford University, United States.
authors.forename: William
authors.initials: W
authors.lastname: Berquist
authors.affiliation: National Urea Cycle Disorders Foundation, United States.
authors.forename: Cynthia
authors.initials: C
authors.lastname: Le Mons
authors.affiliation: Hyperion Therapeutics, Inc., United States.
authors.forename: Ingrid
authors.initials: I
authors.lastname: Rudolph-Angelich
authors.affiliation: Hyperion Therapeutics, Inc., United States.
authors.forename: Marty
authors.initials: M
authors.lastname: Porter
authors.affiliation: Hyperion Therapeutics, Inc., United States.
authors.forename: Bruce F
authors.initials: BF
authors.lastname: Scharschmidt
authors.affiliation: Hyperion Therapeutics, Inc., United States.
authors.forename: Masoud
authors.initials: M
authors.lastname: Mokhtarani
keywords: Dietary management; FAO, Food and Agriculture Organization; GPB, glycerol phenylbutyrate; Inherited metabolic disorders; NHANESs, National Health and Nutrition Examination Surveys; NaPBA, sodium phenylbutyrate; OTC, ornithine transcarbamylase; Phenylbutyrate; Protein intake; RDA, Recommended Daily Allowances; UCD, urea cycle disorder; Urea cycle disorders; WHO, World Health Organization