Down syndrome and Alzheimer disease: response to donepezil

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

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

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

Academic medical center.

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

Oral administration of donepezil for a 5-month period.

The Down Syndrome Dementia Scale.

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

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.