Pharmacologic treatment of seven dystonia is often unsatisfactory. The atyp
ical antipsychotic medication clozapine appears to improve tardive dystonia
associated with conventional neuroleptic use. We studied the efficacy of c
lozapine for severe dystonia in five patients in an open trial. The patient
cohort included four with generalized dystonia and one with Meige syndrome
. All patients were evaluated at baseline and at least weekly while on medi
cation with subjective assessment of response by the patient and physician
rating using the Burke-Fahn-Marsden Evaluation Scale for Dystonia. All five
subjects had significant improvement detected by the Burke-Fahn-Marsden Ev
aluation Scale as well as subjective improvement while on clozapine. Side e
ffects, such as sedation and orthostatic hypotension, developed in all pati
ents but was only treatment-limiting in one subject who developed persisten
t symptomatic orthostatic hypotension and tachycardia. Two of the four rema
ining patients continued clozapine after completion of the study; an additi
onal patient was uncertain if the benefit outweighed the side effects, One
patient discontinued treatment because of difficulty obtaining the FDA-requ
ired weekly white blood cell counts for patients on clozapine. We conclude
that clozapine appears to be effective for generalized and refractory focal
dystonia although its use may be limited by the side effects and need for
hematologic monitoring.