Ocular myasthenia: diagnostic and treatment recommendations and the evidence base

PURPOSE OF REVIEW: Increasing rigor is being applied to medical decision making, but myasthenia gravis, commonly considered the best understood autoimmune disorder from a pathophysiological basis, lags other disciplines in the evidence base used to make clinical decisions. The review attempts to provide a focused, practical guideline for the diagnosis and treatment of ocular myasthenia within the limits of largely retrospective case series and expert opinion. RECENT FINDINGS: Confirmation of clinical diagnosis continues to be challenging for ocular myasthenia. Despite the recognition of a new autoantigen, the muscle-specific kinase protein in generalized myasthenia gravis, it has been found to be only rarely identified in ocular myasthenia patients and therefore the majority of patients lack detectable autoantibodies and confirmation of a neuromuscular transmission disorder relies on specialized testing of single-fiber electromyography. The visual compromise of ocular myasthenia responds poorly to nonpharmacological and cholinesterase inhibitor therapy, and although corticosteroids are thought to be extremely effective, their toxicity is poorly defined in ocular myasthenia patients and whether they reduce the risk of development of generalized disease is not known. SUMMARY: Rigorous clinical trials or large databases with outcome assessments are necessary in order to allow development of rational treatment strategies.