IncobotulinumtoxinA Efficacy/Safety in Upper-Limb Spasticity in Pediatric Cerebral Palsy: Randomized Controlled Trial
Jazyk angličtina Země Spojené státy americké Médium print-electronic
Typ dokumentu klinické zkoušky, fáze III, časopisecké články, randomizované kontrolované studie, práce podpořená grantem
PubMed
34339951
DOI
10.1016/j.pediatrneurol.2021.05.014
PII: S0887-8994(21)00108-9
Knihovny.cz E-zdroje
- Klíčová slova
- Botulinum neurotoxin A, Cerebral palsy, IncobotulinumtoxinA, Multipattern treatment, Pediatric, Spasticity,
- MeSH
- botulotoxiny typu A aplikace a dávkování škodlivé účinky farmakologie MeSH
- dítě MeSH
- dvojitá slepá metoda MeSH
- hodnocení výsledků zdravotní péče MeSH
- horní končetina patofyziologie MeSH
- lidé MeSH
- mladiství MeSH
- mozková obrna komplikace farmakoterapie MeSH
- nervosvalové látky aplikace a dávkování škodlivé účinky farmakologie MeSH
- předškolní dítě MeSH
- svalová spasticita farmakoterapie etiologie patofyziologie MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- klinické zkoušky, fáze III MeSH
- práce podpořená grantem MeSH
- randomizované kontrolované studie MeSH
- Názvy látek
- botulotoxiny typu A MeSH
- incobotulinumtoxinA MeSH Prohlížeč
- nervosvalové látky 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
Citace poskytuje Crossref.org
ClinicalTrials.gov
NCT02002884
