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.