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.