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Should Ocular Myasthenia Gravis Be Treated with Immunomodulation?
The two fundamental treatment questions have not been adequately answered by published studies.
Whether early treatment with immunomodulating therapy is needed for ocular myasthenia gravis (OMG) remains an undecided issue. This comprehensive review of the literature strongly indicates that the two fundamental questions regarding OMG treatment — whether it improves ocular symptoms and whether it reduces the risk for generalization — have not been adequately answered by the studies published to date.
In general, the quality of the published data is low due to an absence of controls, randomization, masking, adequate sample size, or adequate follow-up. Although the data suggest that either azathioprine or corticosteroid therapy reduces diplopia and appears to delay, if not prevent, deterioration to generalized myasthenia gravis (GMG), none of the studies would meet the current standards for prospective clinical trial design and data collection. Up to 60% of patients with OMG progress to GMG, but the authors appropriately call for caution before placing patients on therapies that can have significant systemic adverse effects, except when the expected benefit would be substantial.
Comment: Published studies strongly suggest that prednisone delays or suppresses the onset of GMG in patients with OMG, particularly those at high risk due to abnormal acetylcholine receptor binding antibody. Even accounting for missing data and the unmasked nature of the studies, diplopia in essential directions of gaze seems to be reduced by prednisone and azathioprine, but not by pyridostigmine in most patients.
Do we need a prospective clinical trial to provide a definitive answer on these issues? There are elementary but compelling questions that can be answered only with proper prospective data collection: Is reduction in visual disability enough to justify the risks of these therapies? What are the actual risks of the treatments in this population? Is low-dose treatment effective, and does it carry a low major side-effect profile? For now, each neurologist must individually decide whether to use prednisone or an immunosuppressant (e.g., azathioprine or mycophenolate mofetil), alone or in some combination, and must try not to either under- or overtreat OMG.
— Mark J. Kupersmith, MD
Dr. Kupersmith is Professor of Neurology and Ophthalmology, Albert Einstein School of Medicine, and Director of Neuro-Ophthalmology, Neuro-Ophthalmology Service, Roosevelt Hospital and New York Eye and Ear Infirmary, New York City.
Published in Journal Watch Neurology July 3, 2007
Citation(s):
Benatar M and Kaminski HJ. Evidence report: The medical treatment of ocular myasthenia (an evidence-based review): Report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology 2007 Jun 12; 68:2144-9.
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