Bruk av botulinumtoksin A til barn med cerebral parese

BAKGRUNN Intramuskulære injeksjoner med botulinumtoksin A har i over 20 år vært en etablert behandling av spastisitet hos barn med cerebral parese. Vi undersøkte hvor mange barn med cerebral parese i Norge som får slik behandling, og hvilke retningslinjer som gjelder for behandlingen. MATERIALE OG METODE Data fra femårsregistreringen til Cerebral pareseregisteret i Norge ble benyttet til å undersøke hvor mange barn med cerebral parese født i perioden 1999–2010 som var blitt behandlet med botulinumtoksin A, og om andelen behandlede barn varierte mellom habiliteringssentrene. I en nettbasert spørreundersøkelse kartla vi hvilke retningslinjer for behandlingen alle 21 habiliteringssentrene fulgte. RESULTATER I alt 1 414 barn (gjennomsnittsalder 6,3 år) ble inkludert, hvorav 775 (55 %) var behandlet med botulinumtoksin A. Mellom habiliteringssentrene var det stor variasjon i andelen barn som fikk behandling (38–80 %; p < 0,001). Maksimum dose botulinumtoksin A per behandling per pasient var 200–600 enheter Botox. Fem sentre oppga at de har skriftlige retningslinjer for behandlingsindikasjon. FORTOLKNING Andelen barn med cerebral parese som behandles med botulinumtoksin A varierer betydelig mellom habiliteringssentrene i Norge

The WE-Study: does botulinum toxin A make walking easier in children with cerebral palsy?: Study protocol for a randomized controlled trial

Background Intramuscular injections of botulinum toxin A (BoNT-A) have been a cornerstone in the treatment of spasticity for the last 20 years. In Norway, the treatment is now offered to two out of three children with spastic cerebral palsy (CP). However, despite its common use, the evidence for its functional effects is limited and inconclusive. The objective of this study is to determine whether BoNT-A makes walking easier in children with CP. We hypothesize that injections with BoNT-A in the calf muscles will reduce energy cost during walking, improve walking capacity, increase habitual physical activity, reduce pain and improve self-perceived performance and satisfaction. Methods/design This randomized, double-blinded, placebo-controlled, multicenter trial is conducted in a clinical setting involving three health regions in Norway. Ninety-six children with spastic CP, referred for single-level injections with BoNT-A in the calf muscles, will be invited to participate. Those who are enrolled will be randomized to receive either injections with BoNT-A (Botox®) or 0.9% saline in the calf muscles. Stratification according to age and study center will be made. The allocation ratio will be 1:1. Main inclusion criteria are (1) age 4 − 17.5 years, (2) Gross Motor Function Classification System levels I and II, (3) no BoNT-A injections in the lower limbs during the past 6 months and (4) no orthopedic surgery to the lower limbs during the past 2 years. The outcome measures will be made at baseline and 4, 12 (primary endpoint) and 24 weeks after injections. Primary outcome is change in energy cost during walking. Secondary outcomes are change in walking capacity, change in activity, perceived change in performance and satisfaction in mobility tasks, and pain. The primary analysis will use a linear mixed model to test for difference in change in the outcome measures between the groups. The study is approved by the Regional Ethical Committee and The Norwegian Medicines Agency. Recruitment started in September 2015. Discussion The evaluation of effect is comprehensive and includes objective standardized tests and measures on both impairment and activity level. Results are to be expected by spring 2019