Dataset Information

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: 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.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).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.GAS.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.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.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.ClinicalTrials.gov identifier: NCT01020500.

SUBMITTER: Turner-Stokes L

PROVIDER: S-EPMC3612778 | biostudies-literature | 2013 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

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.

Turner-Stokes Lynne L Fheodoroff Klemens K Jacinto Jorge J Maisonobe Pascal P Zakine Benjamin B

BMJ open 20130318 3

<h4>Objectives</h4>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 ...[more]

PMID: 23512837

Similar Datasets

Project description:OBJECTIVES:Describe the rationale and protocol for the Upper Limb International Spasticity (ULIS)-III study, which aims to evaluate the impact of integrated spasticity management, involving multiple botulinum toxin A (BoNT-A) injection cycles and concomitant therapies, on patient-centred goal attainment. Outline novel outcome assessment methods for ULIS-III and report initial evaluation data from goal setting in early stages of the study. DESIGN:Large international longitudinal cohort study of integrated upper limb spasticity management, including BoNT-A. PARTICIPANTS AND SETTING:ULIS-III is a 2-year study expected to enrol >1000 participants at 58 study centres across 14 countries. INTERVENTIONS:The study design is non-interventional and intended to reflect real-life clinical practice. It will describe injection practices and additional treatment strategies, and record clinical decision-making in a serial approach to long-term spasticity management. OUTCOME MEASURES:ULIS-III will use a goal-directed approach to selection of targeted standardised measures to capture the diversity of presentation, goals and outcomes. ULIS-III will implement the Upper Limb Spasticity Index, a battery of assessments including a structured approach to goal attainment scaling (Goal Attainment Scaling-Evaluation of Outcomes for Upper Limb Spasticity tool), alongside a limited set of standardised measures, chosen according to patients' selected goal areas. Concomitant therapy inputs, patient satisfaction with engagement in goal setting, health economic end points and health-related quality of life data will also be captured. RESULTS OF INITIAL EVALUATION OF GOAL QUALITY:Recruitment started in January 2015. By June 2015, 58 sites had been identified and initial data collected for 79 patients across 13 sites in 3 countries. Goal setting data were quality-checked and centres rated on the basis of function-related and Specific, Measurable, Achievable, Realistic, Timed (SMART) characteristics of goal statements. Overall, 11/13 centres achieved the highest rating (A++). CONCLUSIONS:ULIS-III will provide valuable information regarding treatment of and outcomes from real-life upper limb spasticity management worldwide. TRIAL REGISTRATION NUMBER:NCT02454803; Pre-results.

Project description:ObjectiveTo describe real-life practice and person-centred outcomes in the treatment of poststroke upper limb spasticity with botulinum toxin A (BoNT-A).DesignObservational, prospective study.Setting84 secondary care centres in 22 countries.Participants456 adults (≥18 years) with poststroke upper limb spasticity treated with one cycle of BoNT-A.Methods/outcomesMuscle selection, BoNT-A preparation, injection technique and timing of follow-up were conducted according to routine practice for each centre.Primary outcomeachievement of the patient's primary goal for treatment using goal-attainment scaling (GAS). Measurements of spasticity, standardised outcome measures and global benefits were also recorded.ResultsThe median number of injected muscles was 5 (range 1-15) and the most frequently injected muscles were the long finger flexors, followed by biceps and brachioradialis. The median (range) follow-up time was 14 (2.6 to 32.3) weeks. The common primary treatment goals were passive function (132 (28.9%)), active function (104 (22.8%)), pain (61 (13.4%)), impairment (105 (23%)), involuntary movement (41 (9%)) and mobility (10 (2.2%)). Overall, 363 (79.6%) (95% CI 75.6% to 83.2%) patients achieved (or overachieved) their primary goal and 355 (75.4%) (95% CI 71.2% to 79.2%) achieved their secondary goal. Mean (SD) change from baseline in GAS T-scores was 17.6 (11.0) (95% CI 16.4 to 18.8; p<0.001). GAS T-scores were strongly correlated with global benefit and other standard measures (correlations of 0.38 and 0.63, respectively; p<0.001).ConclusionsBoNT-A demonstrated a clinically significant effect on goal attainment for the real-life management of upper-limb spasticity following stroke. The study confirms the feasibility of a common international data set to collect systematic prospective data, and of using GAS to capture person-centred outcomes relating to passive and active functions and to pain.RegistrationClinicalTrials.gov identifier: NCT01020500.

Project description:ObjectivesThe therapeutic effects of botulinum neurotoxin (BoNT) are well documented in upper limb spasticity. However, several factors may influence treatment efficacy, including targeting of neuromuscular junctions (NMJs). We examined whether NMJ-targeted BoNT injections were non-inferior, in terms of efficacy, to current injection practices.DesignOpen-label prospective evaluator-blinded study.SettingConducted across 20 medical centres in Denmark, Finland, Norway and Sweden (24 September 2012 to 11 March 2015).ParticipantsAged ˃18 years with upper limb spasticity (Modified Ashworth Scale [MAS] score of 2 or 3) following stroke or traumatic brain injury, had received ≥2 consecutive BoNT-A treatment cycles (the latest of which was abobotulinumtoxinA [aboBoNT-A]) and needed BoNT-A retreatment (same modality as previous cycle). Patients requiring aboBoNT-A doses >800units were excluded. In total, 88 patients were randomised (intention-to-treat [ITT] population), most were male (n=58/88, 65.9%) and 54/88 (61.4%) completed the study (per protocol [PP] population).InterventionsRandomisation (1:1) to receive a single dose of aboBoNT-A (≤800 U) according to either current clinical practice (300 U/mL) or as an NMJ-targeted injection (100 U/mL).Primary outcome measureProportion of patients with a ≥1 level reduction from baseline in MAS score at week 4 post-injection (responders).ResultsIn the ITT population, the proportion of responders at elbow flexors was 72.7% in the current practice group and 56.8% in the NMJ-targeted group (adjusted difference -0.1673 [95% CIs: -0.3630 to 0.0284]; p=0.0986). Similar results were observed in the PP population (69.0% vs 68.0%, respectively, adjusted difference 0.0707 [-0.1948 to 0.3362]; p=0.6052).ConclusionsOwing to the limited number of participants, non-inferiority of NMJ-targeted injections could not be determined. However, there was no statistical difference between groups. Larger studies are needed confirm whether the two techniques offer comparable efficacy.Trial registration numberNCT01682148.

Dataset Information

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.

Publications

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.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets