Background: Continuous infusion of intrathecal (IT) baclofen is a high
ly effective standard therapy for severe spasticity of spinal origin.
By contrast, there is limited clinical experience regarding the use of
IT baclofen in treating patients with dystonia, and little is known r
egarding the indications for treatment, efficacy, and safety of IT bac
lofen in this disorder. Objective: To study retrospectively the effect
s of IT baclofen in treating 25 patients with severe segmental or gene
ralized dystonia. Setting: Neurological Institute, Columbia-Presbyteri
an Medical Center, New York, NY. Patients: Twenty-five patients with s
evere segmental or generalized dystonia that was refractory to oral me
dications underwent IT baclofen test dosing. In addition to dystonia,
17 patients had spasticity or painful spasms. Thirteen of 25 patients
responded to the test doses of IT baclofen, according to unblinded neu
rological assessments that included the patient's subjective report; a
ll 13 underwent implantation of a pump for continuous IT baclofen infu
sion. Results: In contrast to reports of patients with spasticity of s
pinal origin, those with dystonia in the present series had a lower re
sponse rate to bolus IT baclofen doses and a smaller degree of clinica
l improvement. For 10 of the 13 responders to the test doses of IT bac
lofen, dystonia rating scale scores of videotaped examinations by blin
ded observers detected no significant change (P<.07) in severity of dy
stonia. Retrospective data from 11 of 13 patients with implantable pum
ps, followed up for a mean interval of 21 months after pump insertion,
showed continuing efficacy in 6 individuals (55%), based on a determi
nation of patient satisfaction; however, only 3 patients (27%) reporte
d a sustained improvement in functional capacity. Five (38%) of the 13
patients with implantable pumps experienced severe complications that
required hospitalization. Conclusions: Despite recent reports that ha
ve described the benefit in small numbers of patients with dystonia, w
e concluded that the role of IT baclofen in treating severe dystonia r
emains uncertain. Intrathecal baclofen may be more effective when dyst
onia is associated with spasticity or pain. In the present series, we
detected no significant difference in the response to IT baclofen in p
atients with or without spasticity or pain, perhaps owing to the small
sample size.