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

Authors


  • Disclosures: Editorial assistance was provided by Hilary Bennett MSc of PAREXEL, and was also supported by Eisai Inc. and Pfizer Inc. Dr. Kishnani has received grants and/or honoraria from various companies including Pfizer, Eisai, Roche, and Novartis. Mr. Heller has received grants and/or honoraria from various companies including Pfizer, Eisai, Roche, and Jannsen. Dr. Spiridigliozzi has received honoraria from various companies including Pfizer, Roche, and Eisai. Dr. Lott is an employee of UC Irvine and has no further disclosures. Dr. Escobar is an employee of Peyton Manning Children's Hospital and has no further disclosures. Dr. Richardson is a current employee of Eisai Inc. Dr. Zhang is a current employee of Pfizer Inc. Dr. McRae is a current employee of Pfizer Inc.

  • How to Cite this Article: Kishnani PS, Heller JH, Spiridigliozzi GA, Lott I, Escobar L, Richardson S, Zhang R, McRae T. 2010. Donepezil for treatment of cognitive dysfunction in children with Down syndrome aged 10–17. Am J Med Genet Part A 152A:3028–3035.

Abstract

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 < 0.0001), with no significant between-group differences. This trial failed to demonstrate any benefit for donepezil versus placebo in children and adolescents with DS, although donepezil appeared to be well tolerated. © 2010 Wiley-Liss, Inc.

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