Journal
MOLECULAR GENETICS AND METABOLISM REPORTS
Volume 25, Issue -, Pages -Publisher
ELSEVIER
DOI: 10.1016/j.ymgmr.2020.100635
Keywords
Children with Pompe disease; Behavior checklists; Screening for behavior problems; School functioning; Emotional functioning; Social functioning
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Funding
- Sanofi Genzyme (Cambridge, Massachusetts, USA)
- Lysosomal Disease Network (LDN)
- National Center for Advancing Translational Sciences (NCATS) Rare Diseases Clinical Research Network (RDCRN) [2U54NS065768-06]
- NCATS at the NIH
- NINDS at the NIH
- NIDDK at the NIH
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Purpose: To improve our understanding of the behavioral, social, and emotional functioning of children and adolescents with Pompe disease. Method: Parents/guardians of 21 children (age 5-18y) with infantile (IPD) or late-onset (LOPD) Pompe disease on long-term enzyme replacement therapy completed three standardized checklists regarding their child's behavior: the Child Behavior Checklist (CBCL), Conners 3 Parent (Conners-3), Behavior Rating Inventory of Executive Function-2 (BRIEF2), and a survey of their child's educational services. Results: Descriptive statistics were used to summarize the findings for each behavior checklist. Age standard scores from each checklist were reported for the IPD (n = 17, 9 females, mean age = 9y, 4 mo; SD = 3y, 8mo) and LOPD (n = 4, 1 female; mean = 11y, 2mo; SD = 2y, 1 mo) groups. The majority of children with Pompe exhibited age-appropriate behavior and emotional functioning on these standardized checklists. However, negative mood symptoms, learning problems, decreased participation in structured social activities, and attentional difficulties were more frequently reported in children with IPD in comparison to same-aged peers. Parents of children with LOPD reported fewer problematic behaviors but endorsed negative mood symptoms and difficulties with peer relations. Most children received accommodations in regular education classrooms at school. Conclusions: These standardized behavior checklists are useful screening tools for the early identification and treatment of behavior, emotional, and social concerns in children with Pompe disease.
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