Upper limb international spasticity study: rationale and protocol for a large, international, multicentre prospective cohort study investigating management and goal attainment following treatment with botulinum toxin A in real-life clinical practice

Abstract

Objectives This article provides an overview of the Upper Limb International Spasticity (ULIS) programme, which aims to develop a common core dataset for evaluation of real-life practice and outcomes in the treatment of upper-limb spasticity with botulinum toxin A (BoNT-A). Here we present the study protocol for ULIS-II, a large, international cohort study, to describe the rationale and steps to ensure the validity of goal attainment scaling (GAS) as the primary outcome measure. Methods and analysis design An international, multicentre, observational, prospective, before-and-after study, conducted at 84 centres in 22 countries across three continents. Participants 468 adults presenting with poststroke upper limb spasticity in whom a decision had already been made to inject BoNT-A (5–12 consecutive participants recruited per centre). Interventions Physicians were free to choose targeted muscles, BoNT-A preparation, injected doses/technique and timing of follow-up in accordance with their usual practice and the goals for treatment. Primary outcome measure: GAS. Secondary outcomes: Measurements of spasticity, standardised outcome measures and global benefits. Steps to ensure validity included: (1) targeted training of all investigators in the use of GAS; (2) within-study validation of goal statements and (3) establishment of an electronic case report form with an in-built tracking facility for separation of baseline/follow-up data. Analysis Efficacy population: all participants who had (1) BoNT-A injection and (2) subsequent assessment of GAS. Primary efficacy variable: percentage (95% CI) achievement of the primary goal from GAS following one BoNT-A injection cycle. Ethics and dissemination This non-interventional study is conducted in compliance with guidelines for good pharmacoepidemiology practices. Appropriate ethical approvals were obtained according to local regulations. ULIS-II will provide important information regarding treatment and outcomes from BoNT-A in real-life upper limb spasticity management. The results will be published separately. Registration ClinicalTrials.gov identifier: NCT01020500.