Dataset Information

Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial.

ABSTRACT:

Background

This is the first large study of onabotulinumtoxinA as treatment for pediatric upper limb spasticity.

Objective

Evaluate efficacy and safety of a single treatment with onabotulinumtoxinA plus occupational therapy (OT).

Methods

In this registrational phase III, multinational study (NCT01603602), participants were randomized 1:1:1 to onabotulinumtoxinA 3 U/kg/OT, 6 U/kg/OT, or placebo/OT. Primary endpoint was average change from baseline at weeks 4 and 6 in Modified Ashworth Scale-Bohannon (MAS) score. Secondary endpoints included Modified Tardieu Scale (MTS), Clinical Global Impression of Change (CGI) and functional Goal Attainment Scale (GAS).

Results

235 participants were randomized. At weeks 4 and 6, onabotulinumtoxinA groups had greater mean reductions in MAS (both -1.9; p < 0.001) versus placebo (-1.2). OnabotulinumtoxinA doses improved dynamic tone per MTS. Mean CGI at weeks 4 and 6 was unchanged in the overall population, but improved in a post hoc analysis of patients with a single affected upper limb (UL) muscle group (elbow or wrist). GAS score for passive goals was significantly higher for 6 U/kg versus placebo at week 12. Most AEs were mild/moderate in severity; overall incidence was similar between groups.

Conclusions

OnabotulinumtoxinA (3 and 6 U/kg) was safe and effective in reducing upper limb spasticity in pediatric participants.

SUBMITTER: Dimitrova R

PROVIDER: S-EPMC8673521 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature

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Publications

Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial.

Dimitrova Rozalina R McCusker Emily E Gormley Mark M Fehlings Darcy D Alter Katharine E KE Greaves Susan S Liu Chengcheng C Brin Mitchell F MF

NeuroRehabilitation 20210101 3

<h4>Background</h4>This is the first large study of onabotulinumtoxinA as treatment for pediatric upper limb spasticity.<h4>Objective</h4>Evaluate efficacy and safety of a single treatment with onabotulinumtoxinA plus occupational therapy (OT).<h4>Methods</h4>In this registrational phase III, multinational study (NCT01603602), participants were randomized 1:1:1 to onabotulinumtoxinA 3 U/kg/OT, 6 U/kg/OT, or placebo/OT. Primary endpoint was average change from baseline at weeks 4 and 6 in Modifie ...[more]

PMID: 34334431

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Project description:IntroductionOnabotulinumtoxinA treatment for spasticity is dependent on numerous factors and varies according to selected treatment goals.ObjectiveTo examine real-world onabotulinumtoxinA treatment utilization and effectiveness in patients with upper limb spasticity over 2 years from the Adult Spasticity International Registry (ASPIRE) study.DesignMulticenter, prospective, observational registry (NCT01930786).SettingFifty-four international clinical sites in North America, Europe, and Asia.PatientsAdults (naïve or non-naïve to botulinum toxins for spasticity) with upper limb focal spasticity related to upper motor neuron syndrome across multiple etiologies.InterventionsOnabotulinumtoxinA administered at clinician's discretion.Main outcome measuresOnabotulinumtoxinA utilization, clinician and patient satisfaction.ResultsFour hundred eighty-four patients received ≥1 treatment of onabotulinumtoxinA for upper limb spasticity. Patients were on average 55.1 years old, 50.8% male, predominantly Caucasian (72.3%), and 38.6% were naïve to botulinum toxins. Stroke was the most frequently reported underlying etiology (74.0%). Most patients (81.2%) had moderate to severe spasticity at baseline. The most commonly treated upper limb clinical presentation was clenched fist (79.1% of patients). Across all presentations, onabotulinumtoxinA doses ranged between 5-600U. Electromyography (EMG) was most often utilized to localize muscles (≥57.0% of treatment sessions). Clinicians (92.9% of treatment sessions) and patients (85.7%) reported being extremely satisfied/satisfied that treatment helped manage spasticity, and clinicians (98.6%) and patients (92.2%) would definitely/probably continue onabotulinumtoxinA treatment. One hundred seventy-nine patients (37.0%) reported 563 adverse events (AEs); 15 AEs in 14 patients (2.9%) were considered treatment related. Sixty-nine patients (14.3%) reported 137 serious AEs; 3 serious AEs in 2 patients (0.4%) were considered treatment related. No new safety signals were identified.ConclusionsASPIRE captured the real-world individualized nature of onabotulinumtoxinA utilization for upper limb spasticity over 2 years, with consistently high clinician- and patient-reported satisfaction. Data in this primary analysis will guide clinical use of onabotulinumtoxinA, as well as provide insights to improve educational programs on spasticity management.

Project description:IntroductionOnabotulinumtoxinA treatment for spasticity varies according to numerous factors and is individualized to meet treatment goals.ObjectiveTo explore real-world onabotulinumtoxinA utilization and effectiveness in patients with lower limb spasticity from the Adult Spasticity International Registry (ASPIRE) study.DesignTwo-year, multicenter, prospective, observational registry (NCT01930786).SettingFifty-four international clinical sites.PatientsAdults (naïve or non-naïve to botulinum toxin[s] treatment for spasticity, across multiple etiologies) with lower limb spasticity related to upper motor neuron syndrome.InterventionsOnabotulinumtoxinA administered at the clinician's discretion.Main outcome measuresOnabotulinumtoxinA treatment utilization, clinician- and patient-reported satisfaction.ResultsIn ASPIRE, 530 patients received ≥1 onabotulinumtoxinA treatment for lower limb spasticity (mean age, 52 years; stroke, 49.4%; multiple sclerosis, 20.4%). Equinovarus foot was treated most often (80.9% of patients), followed by flexed knee (26.0%), stiff extended knee (22.5%), and flexed toes (22.3%). OnabotulinumtoxinA doses ranged between 10 and 1100 U across all presentations. Electromyography (EMG) was most commonly used for injection localization (≥41.1% of treatment sessions). Despite low patient response on the satisfaction questionnaire, clinicians (94.6% of treatment sessions) and patients (84.5%) reported satisfaction/extreme satisfaction that treatment helped manage spasticity, and clinicians (98.3%) and patients (91.6%) would probably/definitely continue onabotulinumtoxinA treatment. These data should be interpreted with care. Twenty-one adverse events (AEs) in 18 patients (3.4%) were considered treatment-related. Sixty-seven patients (12.6%) reported 138 serious AEs; 3 serious AEs in two patients (0.4%) were considered treatment-related. No new safety signals were identified.ConclusionsASPIRE provides long-term observational data on the treatment of lower limb spasticity with onabotulinumtoxinA. Real-world data from this primary analysis can help to guide the clinical use of onabotulinumtoxinA to improve spasticity management.

Dataset Information

Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial.

Background

Objective

Methods

Results

Conclusions

Publications

Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial.

Similar Datasets

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