Journal article
Neuropsychiatric Function Improvement in Pediatric Patients with Phenylketonuria
The Journal of pediatrics, v 260, 113526
01 Sep 2023
PMID: 37263523
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
To evaluate effects of sapropterin dihydrochloride on blood phenylalanine (Phe) and symptoms of neuropsychiatric impairment in children and adolescents with phenylketonuria (PKU).
PKU subjects 8-17 years of age (n = 86) were randomized to double-blind treatment with sapropterin (n = 43) or placebo (n = 43) for 13 weeks, then all received open-label sapropterin therapy for an additional 13 weeks. Blood Phe and symptoms of inattention, hyperactivity/impulsivity (Attention-Deficit/Hyperactivity Disorder Rating Scale IV [ADHD RS-IV]), executive functioning (Behavior Rating Inventory of Executive Function), depression (Hamilton Rating Scale for Depression), and anxiety (Hamilton Rating Scale for Anxiety) were assessed.
Following the 13-week randomization phase, the sapropterin and placebo groups had mean changes in blood Phe of −20.9% and +2.9%, respectively. Corresponding least square mean differences in ADHD RS-IV scores were significantly greater for the sapropterin vs the placebo group: Total (−3.2 points, P = .02), Inattention subscale (−1.8 points, P = .04), and Hyperactivity/Impulsivity subscale (−1.6 points, P = .02). Forest plots favored sapropterin treatment over placebo for all ADHD RS-IV and Behavior Rating Inventory of Executive Function indices. There were no significant differences in reported problems with attention or executive function between the 2 groups at baseline or at week 26 following the 13-week open-label treatment period. Anxiety and depression scores did not differ significantly between cohorts at any time. Sapropterin was well tolerated, with a favorable safety profile.
Sapropterin reduced blood Phe and was associated with significant improvement in parent-reported symptoms of inattention, hyperactivity/impulsivity, and executive functioning in children and adolescents with PKU.
ClinicalTrials.gov, NCT01114737. Registered 27 April 2010, https://clinicaltrials.gov/ct2/show/NCT01114737.
Metrics
Details
- Title
- Neuropsychiatric Function Improvement in Pediatric Patients with Phenylketonuria
- Creators
- Mitzie L. Grant - Drexel University, PsychiatryElaina R. Jurecki - BioMarin Pharmaceutical Inc, Novato, CAShawn E. McCandless - University of Colorado, Anschutz Medical Campus and Children's Hospital Colorado, Aurora, COStephen M. Stahl - Department of Psychiatry, University of California San Diego, San Diego, CADeborah A. Bilder - University of UtahAmarilis Sanchez-Valle - Department of Pediatrics, Division of Genetics and Metabolism, University of South Florida, Tampa, FLDavid Dimmock - Creyon Bio Inc, San Diego, CA
- Publication Details
- The Journal of pediatrics, v 260, 113526
- Publisher
- Elsevier
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Psychiatry; Drexel University
- Web of Science ID
- WOS:001035470600001
- Scopus ID
- 2-s2.0-85162853421
- Other Identifier
- 991021860616304721
UN Sustainable Development Goals (SDGs)
This publication has contributed to the advancement of the following goals:
InCites Highlights
Data related to this publication, from InCites Benchmarking & Analytics tool:
- Collaboration types
- Industry collaboration
- Domestic collaboration
- Web of Science research areas
- Pediatrics