Donepezil for treatment of cognitive dysfunction in children with Down syndrome aged 10-17

• Published in: American journal of medical genetics. Part A 2010-12, Vol.152A (12), p.3028-3035
• Main Authors: Kishnani, Priya S., Heller, James H., Spiridigliozzi, Gail A., Lott, Ira, Escobar, Luis, Richardson, Sharon, Zhang, Richard, McRae, Thomas
• Format: Article
• Language: English
• Subjects: Adolescence; Adolescent; Behavior - drug effects; Biological and medical sciences; Caregivers - psychology; Child; Children; Cholinesterase Inhibitors - adverse effects; Cholinesterase Inhibitors - pharmacology; Cholinesterase Inhibitors - therapeutic use; Chromosome aberrations; cognition; Cognition Disorders - drug therapy; Cognition Disorders - physiopathology; Cognitive ability; Communication; Diarrhea - chemically induced; donepezil; Dose-Response Relationship, Drug; Double-Blind Method; Down syndrome; Down Syndrome - drug therapy; Down Syndrome - physiopathology; Down's syndrome; Drug Tolerance; Female; Humans; Indans - adverse effects; Indans - pharmacology; Indans - therapeutic use; Language; Learning - drug effects; Male; Medical genetics; Medical sciences; Neuropsychological Tests; Piperidines - adverse effects; Piperidines - pharmacology; Piperidines - therapeutic use; Severity of Illness Index; Vomiting - chemically induced
• ISSN: 1552-4825; 1552-4833; 1552-4833
• DOI: 10.1002/ajmg.a.33730

The objective of this 10‐week, randomized, double‐blind, placebo‐controlled multicenter study was to assess the efficacy and safety of donepezil for the treatment of cognitive dysfunction exhibited by children with Down syndrome (DS). Intervention comprised donepezil (2.5–10 mg/day) in children (aged 10–17 years) with DS of mild‐to‐moderate severity. The primary measures were the Vineland‐II Adaptive Behavior Scales (VABS‐II) Parent/Caregiver Rating Form (PCRF) the sum of nine subdomain standardized scores and standard safety measures. Secondary measures included the VABS‐II/PCRF scores on the following domains and their respective individual subdomains: Communication (receptive, expressive, and written); Daily Living Skills (personal, domestic, and community); Socialization (interpersonal relationships, play and leisure time, and coping skills), and scores on the Test of Verbal Expression and Reasoning, a subject‐performance‐based measure of expressive language. At baseline, 129 participants were assigned treatment with donepezil or placebo. During the double‐blind phase, VABS II/PCRF sum of the nine subdomain standardized scores, called v‐scores, improved significantly from baseline in both groups (P