Down syndrome and Alzheimer disease: response to donepezil

Arch Neurol. 2002 Jul;59(7):1133-6. doi: 10.1001/archneur.59.7.1133.


Background: Individuals with Down syndrome who develop Alzheimer disease may show an improvement in cognitive functioning after treatment with acetylcholinesterase inhibitors.

Objective: To determine whether individuals with Down syndrome and Alzheimer disease will show improvement after institution of donepezil treatment.

Design: A nonrandomized controlled trial using donepezil in a pilot study format.

Setting: Academic medical center.

Patients: Convenience sample of 6 treated patients with Down syndrome and 9 closely matched historical control subjects.

Intervention: Oral administration of donepezil for a 5-month period.

Primary outcome measure: The Down Syndrome Dementia Scale.

Results: Significant improvement in dementia scores for the treated group during a 3- to 5-month period (P =.03).

Conclusions: Acetylcholinesterase inhibitors may be helpful in reversing the symptoms of dementia during early and middle stages of cognitive decline. These findings support the rationale for a more extensive study of the efficacy of acetylcholinesterase inhibitors in Down syndrome dementia.

Publication types

  • Clinical Trial
  • Controlled Clinical Trial
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Alzheimer Disease / complications
  • Alzheimer Disease / drug therapy*
  • Cholinesterase Inhibitors / administration & dosage
  • Cholinesterase Inhibitors / therapeutic use*
  • Donepezil
  • Down Syndrome / complications
  • Down Syndrome / drug therapy*
  • Drug Administration Schedule
  • Female
  • Humans
  • Indans / administration & dosage
  • Indans / therapeutic use*
  • Male
  • Middle Aged
  • Nootropic Agents / administration & dosage
  • Nootropic Agents / therapeutic use*
  • Pilot Projects
  • Piperidines / administration & dosage
  • Piperidines / therapeutic use*
  • Psychiatric Status Rating Scales
  • Treatment Outcome


  • Cholinesterase Inhibitors
  • Indans
  • Nootropic Agents
  • Piperidines
  • Donepezil