Nutrients · 7 authors, 4 centres
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This 6-month prospective cohort study of children with phenylketonuria found that sapropterin treatment increased natural protein tolerance and reduced caregiver anxiety and burden of care, but did not significantly change children's anxiety/depression scores.
This 6-month prospective cohort study evaluated the impact of sapropterin dihydrochloride on diet quality and burden of care in children with phenylketonuria. The study found that natural protein intake at least doubled after 6 months of sapropterin treatment without loss of metabolic control, and there was a decreased dependence on special low protein foods. Caregiver anxiety and depression levels were significantly lower while children were taking sapropterin, and caregivers reported alleviated familial-social impact and personal strain. However, no significant changes were observed in the patients' own anxiety or depression scores. The authors note that while protein intake increased, the quality of nutritional intake from food was initially inadequate, and changes in food patterns were a complex process requiring careful counseling. The study's limitations include a small sample size and the use of non-PKU-specific questionnaires, with results representing the first 6 months of a planned 2-year follow-up.
