Levodopa for Dystonia in Children: A Case Series and Review of the Literature.

Background: Levodopa is used to treat hyperkinetic movements in children with dopa-responsive dystonia. However, levodopa may also be helpful in treating other forms of dystonia when used beyond a brief trial period.

Methods: We performed a retrospective review of all children referred to our institution for evaluation of generalized dystonia and subsequently treated with carbidopa-levodopa. Motor function was assessed using video recordings and examination notes, quantified with the Burke-Fahn-Marsden Dystonia Rating Scale.

Results: Long-term treatment with carbidopa-levodopa moderately improved motor function, whereas short-term use did not. Carbidopa-levodopa was well tolerated without untoward effects.

Conclusions: Dystonia is a significant cause of disability with limited effective treatment options. Published work is restricted but generally supports the findings of this review. A well-controlled study to examine the utility of carbidopa-levodopa treatment for dystonia is needed.