Thursday, 3 April 2003

This presentation is part of : Clinical neurosciences in dementia

Donepezil-Treated Alzheimer’s Disease Patients Initially Rated as Showing “No Apparent Clinical Benefit” Demonstrate Significant Benefits When Therapy is Continued: Results From a Randomized, Placebo-Controlled Trial

A Kurz1, M Barcikowska2, S Jakobsen2, C Karageorgiou2, M Lambert2, J McCarthy2, M Trixler2, CJ Wouters2, S Qvitzau2, S Richardson2, E Schwam2, Y Xu2, R Schindler2, and P Johannsen2. (1) Technische Universitat Munchen, Munich, Germany, (2) Poland

Objective: The aim of the donepezil AWARE (Aricept WAshout and REchallenge) study is to determine the nature and extent to which Alzheimer’s disease (AD) patients showing “no apparent clinical benefit” during 12-24 weeks of initial donepezil therapy benefit from continued treatment.

Background: AD patients who do not exhibit improvement are often discontinued from cholinesterase inhibitor therapy. However, due to the nature of AD, these patients may still experience therapeutic benefits. Furthermore, current methods of rating clinical benefit may be biased towards cognition, thus benefits in other domains might not be identified.

Design: AWARE consists of 3 phases: 1) 24-week, pre-randomization, open-label donepezil treatment phase; 2) 12-week, randomized, double-blind, placebo-controlled phase; 3) 12-week, single-blind donepezil treatment phase.

Materials and Methods: Patients with mild to moderate AD were enrolled. In the pre-randomization phase, all patients received donepezil 5 mg/day for 28 days then 10 mg/day. Clinical benefit was assessed at Weeks 12, 18, and 24; at each time point, patients rated as showing “no apparent clinical benefit” were randomized into the double-blind phase. Patients classified as showing “no apparent clinical benefit” were those who exhibited decline or no change from baseline on the MMSE and whose physician was not sufficiently certain of clinical benefit to warrant continued treatment (assessed by formal questionnaire). Efficacy assessments included the ADAS-cog, MMSE, NPI, and DAD. Results are reported from preliminary analyses of the randomized, placebo-controlled, double-blind phase data as least-squares mean change from baseline for Week 12 intent-to-treat observed cases.

Results: 814 patients enrolled and received donepezil for 12-24 weeks. Of these, 202 were classified as showing “no apparent clinical benefit” and were then randomized to continue on donepezil 10 mg/day (n=99) or switch to placebo (n=103). Significant differences in favor of the donepezil group compared to placebo were observed (MMSE treatment difference -1.13, P<0.05; NPI treatment difference 3.16, P<0.05). Donepezil-treated patients remained above baseline on the DAD for 12 weeks, while placebo-treated patients declined (treatment difference -3.67, P=0.NS). Donepezil-treated patients also showed less decline than placebo-treated patients on the ADAS-cog (treatment difference 0.57, P=NS).

Conclusion: Based on criteria requiring evidence of stabilization or decline, a small proportion of donepezil-treated patients were initially classified as showing “no apparent clinical benefit” after 12-24 weeks of therapy. During a further 12-week treatment period, patients in this group who continued on donepezil demonstrated cognitive and behavioral benefits, compared with those who switched to placebo. Physicians should therefore be cautious when deciding whether to discontinue donepezil treatment, as patients who apparently decline when assessed on cognitive or global measures may still experience significant treatment benefits.

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