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.

Figures

Figure 1

World map showing geographical distribution of participating centres in Upper Limb International Spasticity II (ULIS-II).

Figure 2

Flow chart for recruitment.

Figure 3

Converting the goal attainment scaling-light verbal scoring system to a numerical five-point scale (−2 to +2). The verbal descriptions align with how clinicians normally think about and describe goal attainment. They allow goal attainment to be recorded without reference to the numeric scores, and so avoid the perceived negative connotations of zero and minus scores.

Figure 4

(A) Percentage of centres achieving high quality ratings in rounds 1 and 2 during validation of goal attainment scaling statements. See table 1 for description of WHO International Classification of Functioning (ICF) domains A, B and C, and specific, measurable, achievable, realistic and timed (SMART) descriptor ratings ++, + and −. (B) Median quality rating of final goal statements by participating country. Goal quality ratings were derived from the WHO ICF domain rating (A=4, A/B=3, B=2 and C=1) and the SMART rating (‘++’=4, ‘+’=3, ‘+/−’=2 and ‘−’=1). Each centre was assigned two goal quality ratings and the graph shows the medians for each participating country.