IncobotulinumtoxinA Efficacy/Safety in Upper-Limb Spasticity in Pediatric Cerebral Palsy: Randomized Controlled Trial
Language English Country United States Media print-electronic
Document type Clinical Trial, Phase III, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't
PubMed
34339951
DOI
10.1016/j.pediatrneurol.2021.05.014
PII: S0887-8994(21)00108-9
Knihovny.cz E-resources
- Keywords
- Botulinum neurotoxin A, Cerebral palsy, IncobotulinumtoxinA, Multipattern treatment, Pediatric, Spasticity,
- MeSH
- Botulinum Toxins, Type A administration & dosage adverse effects pharmacology MeSH
- Child MeSH
- Double-Blind Method MeSH
- Outcome Assessment, Health Care MeSH
- Upper Extremity physiopathology MeSH
- Humans MeSH
- Adolescent MeSH
- Cerebral Palsy complications drug therapy MeSH
- Neuromuscular Agents administration & dosage adverse effects pharmacology MeSH
- Child, Preschool MeSH
- Muscle Spasticity drug therapy etiology physiopathology MeSH
- Check Tag
- Child MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase III MeSH
- Research Support, Non-U.S. Gov't MeSH
- Randomized Controlled Trial MeSH
- Names of Substances
- Botulinum Toxins, Type A MeSH
- incobotulinumtoxinA MeSH Browser
- Neuromuscular Agents MeSH
BACKGROUND: This randomized phase 3 study with double-blind main period (MP) and open-label extension (OLEX; NCT02002884) assessed incobotulinumtoxinA safety and efficacy for pediatric upper-limb spasticity treatment in ambulant/nonambulant (Gross Motor Function Classification System [GMFCS] I-V) patients, with the option of combined upper- and lower-limb treatment. METHODS: Patients were aged two to 17 years with unilateral or bilateral spastic cerebral palsy (CP) and Ashworth Scale (AS) score ≥2 in treatment-selected clinical patterns. In the MP, patients were randomized (2:1:1) to incobotulinumtoxinA 8, 6, or 2 U/kg body weight (maximum 200, 150, 50 U/upper limb), with optional lower-limb injections in one of five topographical distributions (total body dose ≤16 to 20 U/kg, maximum 400 to 500 U, depending on body weight and GMFCS level). In the OLEX, patients received three further treatment cycles, at the highest MP doses (8 U/kg/upper limb group). Outcomes included AS, Global Impression of Change Scale (GICS), and adverse events (AEs). RESULTS: AS scores improved from baseline to week 4 in all MP dose groups (n = 350); patients in the incobotulinumtoxinA 8 U/kg group had significantly greater spasticity improvements versus the 2 U/kg group (least-squares mean [standard error] for upper-limb main clinical target pattern -1.15 [0.06] versus -0.93 [0.08]; P = 0.017). Investigator's, child/adolescent's, and parent/caregiver's GICS scores showed improvements in all groups. Treatment benefits were sustained over further treatment cycles. AE incidence did not increase with dose or repeated treatment across GMFCS levels. CONCLUSIONS: Data provide evidence for sustained efficacy and safety of multipattern incobotulinumtoxinA treatment in children and adolescents with upper-limb spasticity.
Beaumont Pediatric Physical Medicine and Rehabilitation Royal Oak Royal Oak Michigan
Department of Neurology Collegium Medicum Jagiellonian University Krakow Poland
Merz Pharmaceuticals GmbH Frankfurt am Main Germany
Rady Children's Hospital San Diego California
Shirley Ryan AbilityLab Northwestern Feinberg School of Medicine Chicago Illinois
References provided by Crossref.org
ClinicalTrials.gov
NCT02002884
