Down syndrome and Alzheimer disease: response to donepezil.

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.