Background: Classically, clinical trials are based on the placebo-control design. Our aim was to analyze the placebo effect in Huntington's disease.
Methods: Placebo data were obtained from an international, longitudinal, placebo-controlled trial for Huntington's disease (European Huntington's Disease Initiative Study Group). One-hundred and eighty patients were evaluated using the Unified Huntington Disease Rating Scale over 36 months. A placebo effect was defined as an improvement of at least 50% over baseline scores in the Unified Huntington Disease Rating Scale, and clinically relevant when at least 10% of the population met it.
Results: Only behavior showed a significant placebo effect, and the proportion of the patients with placebo effect ranged from 16% (first visit) to 41% (last visit). Nondepressed patients with better functional status were most likely to be placebo-responders over time.
Conclusions: In Huntington's disease, behavior seems to be more vulnerable to placebo than overall motor function, cognition, and function
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