Long-term Efficacy of Local Doxorubicin Chemomyectomy in Patients with Blepharospasm and Hemifacial Spasm Jonathan D. Wirtschafter, MD, Linda K. McLoon, PhD 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 amelio- rated or the patient requested discontinuation. Main Outcofne 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 supple- mentation. 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 blepharosp&sm 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. Ophthalmology 1998; 105:342-346 Essential blepharospasm and hemifacial spasm of the eye- lids are not uncommon conditions that are often debilitat- ing and cause inability to drive an automobile, read, or engage in social discourse. The etiology of essential blepharospasm is unknown. Hemifacial spasm is usually due to compression of the proximal portion of the facial nerve, and often may be treated by craniotomy with de- Originally received: April 9, 1997. Revision accepted: August 22, 1997. From the Departments of Ophthalmology, Neurology, and Neurosur- gery, University of Minnesota Medical School, Minneapolis Minnesota. Supported in part by unrestricted grants to the Department of Ophthal- mology by Research to Prevent Blindness, Inc., New York, New York, and Minnesota Lions and Lioness Clubs, Minneapolis, Minnesota. The authors have no proprietary interest in any aspect of this work. Reprint requests to Jonathan D. Wirtschafter, MD, Department of Oph- thalmology, University of Minnesota Medical School, 420 Delaware St, SE, Minneapolis, MN 55455-0501. compression of the nerve. This article updates prior re- ports ~'2 on the progress of doxorubicin chemomyectomy for the permanent treatment of essential blepharospasm and hemifacial spasm symptomatically localized to the eyelids, and provides a long-term assessment of the safety and efficacy of this treatment. No new patients have been recruited since'-1994, when we became aware that labora- tory studies indicated that the efficacy and safety of the treatment might be further improved when changes in the human protocol are implemented in the near future. This report also serves to call to the attention of clinicians the research published in journals not usually read by clinical ophthalmologists that discuss the scientific basis for chemomyectomy and that have furthered our understand- ing of the anatomy of the orbicularis oculi muscle. As a result of these studies, new treatment protocols will be tested that may further increase the safety and efficacy of this procedure. Doxorubicin is an antimitotic and antimetabolic drug 342