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Down Syndrome Abstract
of the Month: Dec 2002

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A 24-week, double-blind, placebo-controlled trial of donepezil in patients with Down syndrome and Alzheimer's disease.

Prasher VP, Huxley A, Haque MS; Down syndrome Ageing Study Group.
Int J Geriatr Psychiatry 2002 Mar;17(3):270-8

Monyhull Hospital, Monyhull Hall Road, Kings Norton, Birmingham, UK.

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BACKGROUND: Donepezil Hydrochloride (Aricept) is a selective anticholinesterase inhibitor developed for the treatment of Alzheimer's disease (AD). This study investigated the safety and efficacy of the drug to treat Down syndrome (DS) adults with mild to moderate AD. METHOD: This was a 24-week, double blind, placebo controlled, parallel-group trial. Patients were randomized to receive placebo or donepezil (5 mg per day during the first four weeks, and then 10 mg per day thereafter). Primary efficacy was measured using the Dementia Scale for Mentally Retarded Persons (DMR), and secondary efficacy was measured using the Severe Impairment Battery (SIB), Neuropsychiatric Inventory (NPI) and by the Adaptive Behavior Scale (ABS). RESULTS: A total of 30 DS patients with AD entered the study of which 27 were included in the subsequent data analysis. The donepezil group had non-statistically significant reduction in deterioration in DMR, SIB, and ABS mean scores relative to the placebo group. However NPI scores showed less improvement in the donepezil group when compared to the placebo group. Fifty percent of subjects in the donepezil group showed improvement in mean DMR scores at the end point compared to baseline, when compared to 31% on placebo. There were no life threatening adverse effects associated with treating adults with DS with donepezil. A number of side-effects did occur including diarrhoea, insomnia, fatigue, and nausea. CONCLUSION: Donepezil Hydrochloride administered once a day appears to be generally well tolerated and safe in DS adults who have AD. There is some possible efficacy in the treatment of symptoms of mild to moderate Alzheimer's disease in this population, although the sample size of this study was too small for statistical significance. It is recommended that donepezil, with the appropriate precautions, should be considered for the treatment of AD in adults with DS as deemed by a specialist.

My comments:

For a background on this medication and DS, see the April 1999 article. Just like that study, this one is designed more as a "pilot study" which shows that there is cause to fund a larger study. However, the 1999 study only looked at 4 adults, as opposed to the 27 in the above study.

The good news is that at the present time, Pfizer is funding a large, multi-center study in the US on adults with DS. This study will look for improvements in attention, language, memory and other cognitive areas in the adult subjects. It will take some time before we find out results, as the wheels of research turn slowly, but there is cause for optimism.

We should be hearing soon about the results of the Duke pilot study of cognitive improvements in schoolchildren with DS and donepezil.

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