Dietary Liberalization in Tetrahydrobiopterin-Treated PKU Patients: Does It Improve Outcomes?

Roeland A.F. Evers, Annemiek M.J. van Wegberg, Anita MacDonald, Stephan C.J. Huijbregts, Vincenzo Leuzzi, Francjan J. van Spronsen*

*Corresponding author for this work

    Research output: Contribution to journalReview articlepeer-review

    5 Citations (Scopus)
    79 Downloads (Pure)

    Abstract

    Purpose: this systematic review aimed to assess the effects of dietary liberalization following tetrahydrobiopterin (BH4) treatment on anthropometric measurements, nutritional biomarkers, quality of life, bone density, mental health and psychosocial functioning, and burden of care in PKU patients. Methods: the PubMed, Cochrane, and Embase databases were searched on 7 April 2022. We included studies that reported on the aforementioned domains before and after dietary liberalization as a result of BH4 treatment in PKU patients. Exclusion criteria were: studies written in a language other than English; studies that only included data of a BH4 loading test; insufficient data for the parameters of interest; and wrong publication type. Both within-subject and between-subject analyses were assessed, and meta-analyses were performed if possible. Results: twelve studies containing 14 cohorts and 228 patients were included. Single studies reported few significant differences. Two out of fifteen primary meta-analyses were significant; BMI was higher in BH4-treated patients versus controls (p = 0.02; standardized mean difference (SMD) (95% confidence interval (CI)) = −0.37 (−0.67, −0.06)), and blood cholesterol concentrations increased after starting BH4 treatment (p = 0.01; SMD (CI) = −0.70 (−1.26, −0.15)). Conclusion: there is no clear evidence that dietary liberalization after BH4 treatment has a positive effect on anthropometric measurements, nutritional biomarkers, or quality of life. No studies could be included for bone density, mental health and psychosocial functioning, and burden of care.

    Original languageEnglish
    Article number3874
    Number of pages17
    JournalNutrients
    Volume14
    Issue number18
    DOIs
    Publication statusPublished - Sept-2022

    Keywords

    • biomarkers
    • BMI
    • diet
    • growth
    • mental health
    • meta-analysis
    • nutrition
    • phenylketonuria
    • psychosocial functioning
    • quality of life
    • systematic review
    • tetrahydrobiopterin
    • weight

    Fingerprint

    Dive into the research topics of 'Dietary Liberalization in Tetrahydrobiopterin-Treated PKU Patients: Does It Improve Outcomes?'. Together they form a unique fingerprint.

    Cite this