Approach to the Treatment of Pediatric Dystonia

Abstract

Dystonia is the most common movement disorder in the pediatric population. It can affect normal motor development and cause significant motor disability. The treatment of pediatric dystonia can be very challenging as many children tend to be refractory to standard pharmacological interventions. Pharmacological treatment remains the first-line approach in pediatric dystonia. However, despite the widespread use of different ani-dystonia medications, the literature is limited to small clinical studies, case reports, and experts’ opinions. Botulinum neurotoxin (BoNT) is a well-established treatment in adults with focal and segmental dystonia. Despite the widespread use of BoNT in adult dystonia the data to support its use in children is limited with the majority extrapolated from the spasticity literature. For the last 2 decades, deep brain stimulation (DBS) has been used for a wide variety of dystonic conditions in adults and children. DBS gained increased popularity in the pediatric population because of the dramatic positive outcomes reported in some forms of genetic dystonia and the subsequent consensus that DBS is generally safe and effective. This review summarizes the available evidence supporting the efficacy and safety of pharmacological treatment, BoNT, and DBS in pediatric dystonia and provides practical frameworks for the adoption of these modalities.

Deep Brain Stimulation of the Globus Pallidus Internus in a Child with Refractory Dystonia due to L2-Hydroxyglutaric Aciduria

“…Targeting the GPi for medically resistant pediatric dystonia is becoming the standard of care [14][15][16][17]. This is due to the effectiveness, low complication rates, and the ability to adjust stimulation parameters.…”

Update in Pediatric Neurocritical Care: What a Neurologist Caring for Critically Ill Children Needs to Know

“…Suggested standardized approach for the management of acute status dystonicus. [67][68][69][70][71][72][73] in NMOSD, deficits are often severe at onset and spinal lesions are longitudinally extensive (spanning at least three vertebral bodies). Conus medullaris involvement is thought to be a distinguishing feature.…”

“…Suggested standardized approach for the management of acute status dystonicus. [67][68][69][70][71][72][73] Seminars in Neurology Vol. 44 No.…”

Deep Brain Stimulation for Refractory Status Dystonicus in Children: Multicenter Case Series and Systematic Review

“…This usually results in extended pediatric intensive care unit (PICU) admissions where children may be intubated, ventilated, and given sedating agents or muscle relaxants. 5,7,12 Recognizing the challenges in identifying all patients with SD, a prior study reported that only a small proportion of patients with severe SD respond to first-line pharmacotherapy. 5 These findings highlight the importance of multimodal treatment for SD, especially in cases where it is refractory to medical therapy.…”

“…Pharmacological therapy (including trihexyphenidyl, tetrabenazine, and benzodiazepines) is often not sufficient to resolve SD. This usually results in extended pediatric intensive care unit (PICU) admissions where children may be intubated, ventilated, and given sedating agents or muscle relaxants 5,7,12 . Recognizing the challenges in identifying all patients with SD, a prior study reported that only a small proportion of patients with severe SD respond to first‐line pharmacotherapy 5 .…”