Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).
The investigators will compare intra-subject change in adults with PKU who follow a protein-restricted diet (protein intake less than the RDA for protein based on weight (0.8 g/kg/day) with or without a PKU medical food) within 90 days of starting pegvaliase (baseline) and 9 and 15 months later. The investigators will collect laboratory measures of nutrition status, anthropometrics, Dual-Energy X-Ray Absorptiometry (DXA) to measure body composition and bone mineral density (BMD), and indirect calorimetry to measure resting energy expenditure (REE). In addition, three-day food records will be obtained to assess protein intake and overall diet quality.
Study Type
OBSERVATIONAL
Enrollment
12
Boston Children's Hospital
Boston, Massachusetts, United States
Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in serum markers of protein status at months 0, 9, and 15.
Time frame: 15 months
Intra-subject difference in lean body mass (%LBM) from month 0 to 15.
Time frame: 15 months
Intra-subject difference in bone mineral density from month 0 to 15.
Time frame: 15 months
Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in BMI at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15.
Time frame: 15 months
Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15.
Three-Factor Eating Questionnaire (TFEQ)-18 is an 18-item questionnaire measured on a four-point scale cognitive restraint (6 items), uncontrolled eating (9 items) and emotional eating (3 items). Responses are measured on a four-point scale and the raw scores for each subscale are transformed to correspond to a 0-100 point scale. Higher scores in the respective scales are indicative of greater cognitive restraint, emotional eating, and uncontrolled eating.
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Time frame: 15 months
Intra-subject change in food neophobia at months 0, 9, and 15.
Food Neophobia Scale, which was adapted for a PKU population, includes nine questions measured on a seven-point scale with a higher total score indicative of greater food neophobia (score range 9-63).
Time frame: 15 months
Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15.
Epicurean eating tendencies questionnaire includes seven items measured with a seven-point scale (range 7-49). Higher scores indicative of greater Epicurean eating tendencies.
Time frame: 15 months