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Randomized controlled trial of a protein substitute with prolonged release on the protein status of children with
Marcello Giovannini1, Enrica Riva, Elisabetta Salvatici
1a Department of Paediatrics , San Paolo Hospital, University of Milan , Milan , ITALY.
Insights
A new phenylalanine-free protein substitute with prolonged release may improve protein status in children with phenylketonuria (PKU). This study suggests potential benefits over conventional substitutes for managing PKU protein intake.
Area of Science:
- Biochemistry
- Pediatric Nutrition
- Metabolic Disorders
Background:
- Phenylketonuria (PKU) is a genetic disorder requiring strict dietary management.
- Children with PKU often face challenges in maintaining optimal protein status due to dietary restrictions.
- Conventional protein substitutes are the standard for nutritional management in PKU.
Purpose of the Study:
- To evaluate the efficacy of a novel phenylalanine-free protein substitute with prolonged release.
- To compare the impact of prolonged-release versus conventional protein substitutes on protein status in children with PKU.
- To assess key protein biomarkers including albumin, transthyretin, and retinol-binding protein (RBP).
Main Methods:
- A randomized controlled trial involving 60 children with PKU aged 7-16 years.
- Participants received either a prolonged-release (test) or conventional protein substitute for 30 days.
- Protein status was assessed using serum albumin, transthyretin, and RBP levels.
Main Results:
- No significant differences in anthropometry or macronutrient intake between groups.
- While albumin and RBP remained within normal ranges, transthyretin levels showed improvement in the prolonged-release group.
- Mean transthyretin increased from 19.1 to 20.7 mg/dL in the test group, compared to a minimal change in the conventional group.
Conclusions:
- Prolonged-release phenylalanine-free protein substitutes show potential for improving protein status in children with PKU.
- This novel approach may offer advantages in managing transthyretin levels, a marker of protein insufficiency.
- Further research is warranted to confirm long-term benefits and optimal use in PKU management.
Objective:
To examine whether a phenylalanine-free protein substitute with prolonged release may be beneficial to the protein status of children with phenylketonuria (PKU) compared to conventional substitutes.
Methods:
Sixty children with PKU, 7 to 16 years of age, were randomly allocated to receive either a prolonged-release (test) or the current conventional protein substitute for 30 days. Subjects were additionally sex and age matched with 60 subjects with mild hyperphenylalaninemia and 60 unaffected subjects. The protein status in children with PKU was assessed by albumin, transthyretin, and retinol-binding protein (RBP), and changes throughout the trial period were the primary outcome measures.
Results:
Children with PKU did not differ in anthropometry from children with mild hyperphenylalaninemia or unaffected children but they ingested lower amounts of proteins (p < 0.01). No differences occurred throughout the trial between or within children with PKU who received the test or conventional substitute for macronutrient intake. Albumin and RBP concentrations were within the age-specific reference range for all children. The rate of protein insufficiency (transthyretin concentration less than 20 mg/dL) did not differ statistically between children receiving test or conventional substitute (recruitment 51.8% vs 53.6%; end of the trial 44.4% vs 50.0%) but mean transthyretin recovered over 20 mg/dL in children who received the test substitute, increasing from 19.1 to 20.7 mg/dL (mean change, 1.6 mg/dL; 95% confidence interval 0.4 to 2.8 mg/dL). In children receiving conventional substitute mean transthyretin changed from 19.0 to 19.2 mg/dL (0.2; -0.2 to 0.6) mg/dL.
Conclusions:
Protein substitutes with prolonged release might be beneficial to protein status in children with phenylketonuria.
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