Rationale and design of a multicentre, double-blind, prospective, randomized, European and Canadian study: Evaluating patient outcomes and costs of managing adults with post-stroke focal spasticity

Abstract

Objective: This report describes the design of a study aiming to provide evidence for the extended use of botulinum toxin A (BOTOX®, Allergan Inc.) in focal post-stroke upper and lower limb spasticity and to evaluate the impact of incorporating botulinum toxin A treatment into the rehabilitation of patients with spasticity. Design: international, prospective, randomized, double-blind, placebo-controlled study with an open-label extension. Methods: Approximately 300 adults with a stroke occurring ≥ 3 months before screening, presenting with symptoms and signs of an upper motor neuron syndrome and focal spasticity-related functional impairment, were randomized to botulinum toxin A + standard care or placebo+standard care. Study medication was administered at baseline and again at Week 12 if required, with follow-up to 52 weeks. The primary endpoint was the number of patients who achieved their investigator-rated principal active functional goal (as measured by Goal Attainment Scaling), at 10 weeks after the second injection (Weeks 22-34) or at the 24-week visit if no second injection was administered. Secondary endpoints included changes from baseline in level of goal achievement, health-related quality of life and resource utilization. Conclusion: The BOTOX® Economic Spasticity Trial (BEST) will provide information regarding clinical and costeffectiveness of botulinum toxin + standard care vs standard care alone in patients with upper and/or lower limb poststroke spasticity typically seen in clinical practice. © 2011 The Authors.