Long-term efficacy of local doxorubicin chemomyectomy in patients with blepharospasm and hemifacial spasm.

OBJECTIVE: This study examines the long-term follow-up of all patients treated with doxorubicin injections in the eyelids.
DESIGN: Nonrandomized clinical trial. PARTICIPANTS: Eighteen patients with blepharospasm (12 female; 6 male) and nine patients with hemifacial spasm (4 female; 5 male). INTERVENTION: Eyelids were repeatedly injected at intervals of 10 or more weeks until the spasms were ameliorated or the patient requested discontinuation. MAIN OUTCOME MEASURE: Clinical "cure" defined as sufficient symptomatic relief to defer further paralytic treatment. All patients have been followed for more than 1 year since the last injection.
RESULTS: Nine of 18 patients with blepharospasm completed the full course of treatment and are considered "cured" for more than 1 year (median, 3 years; maximum, 6 years). Six of nine patients with hemifacial spasm completed treatment. Five of these six patients are considered "cures," lasting for more than 4.5 to 6 years. Two additional patients, one with blepharospasm and one with hemifacial spasm, had significant amelioration and were untreated for more than 3 years after the last doxorubicin injection, but occasionally request botulinum toxin supplementation. The minimum effective dose per treated eyelid ranged from 1.0 to 4.2 mg (median, 2.25 mg). The treatment-related discontinuations and complications were related to skin inflammation. Four of the 14 "cured" patients required some surgical "touch-up" on 1 eyelid. However, all the patients who completed treatment are either cured or have had significant amelioration of symptoms.
CONCLUSIONS: Doxorubicin chemomyectomy is an evolving technique and an effective treatment for essential blepharospasm and hemifacial spasms symptomatically localized to the eyelids. Sixteen (59%) of the initial series of 27 patients completed the treatment. Of these, all are apparently cured or their symptoms significantly ameliorated. In the future, an even higher proportion would be expected to complete the treatment due to improvements in the selection criteria and treatment protocols developed during this 8-year trial. While the treatment appears to be reasonably safe compared with surgical myectomy in its present form, the authors are continuing to explore and introduce additional cotreatments to minimize the acute skin changes and maximize the long-term effectiveness of the myectomy.