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Baclofen in the treatment of idiopathic dystonia in children
1Dystonia Clinical Research Center, Columbia Presbyterian Medical Center, New York, New York.
Insights
Baclofen shows promise for treating childhood idiopathic dystonia. Early treatment initiation appears key for sustained symptom improvement in pediatric patients.
Area of Science:
- Neurology
- Pediatric Neurology
Background:
- Idiopathic dystonia is a neurological movement disorder affecting children.
- Baclofen is a muscle relaxant used for adult-onset dystonia, but its efficacy in pediatric idiopathic dystonia is less documented.
Observation:
- A study evaluated baclofen treatment in 16 pediatric patients (under 21 years) with idiopathic dystonia.
- Five patients experienced substantial improvement, two had moderate improvement, and nine showed no benefit.
Findings:
- Three patients with prior transient improvement on anticholinergics showed sustained, dramatic response with added baclofen.
- Sustained improvement was observed in five patients for a mean of 3.8 years on baclofen.
- Shorter symptom duration before baclofen therapy correlated with better treatment response (p < .002).
Implications:
- Baclofen can be an effective therapeutic option for childhood idiopathic dystonia.
- Early intervention with baclofen may lead to more significant and sustained symptom relief.
- Further research into baclofen's role in pediatric movement disorders is warranted.
Abstract:
Baclofen benefits some patients with adult onset dystonia, but few reports document the response to baclofen of children with idiopathic dystonia. Sixteen of 80 patients less than age 21 years with idiopathic dystonia seen by the Movement Disorder Group at Columbia-Presbyterian Medical Center in New York were treated with baclofen. Five had substantial improvement in symptoms, two had moderate improvement, and nine failed to benefit. Three of the improved patients had transient improvement on high dose anticholinergics, but had sustained, dramatic improvement when baclofen was added. At last follow-up, five patients maintained improvement for a mean 3.8 years (19 months-8 years) on a mean 79 mg of baclofen (40-120 mg). Response to baclofen did not correlate with age at onset of dystonia or age at onset of therapy, but did correlate with duration of symptoms before therapy (3 years for those who improved vs 7.8 years for those who did not: p less than .002 by t-test). Baclofen can be an effective treatment for childhood dystonia.