New Treatments in Neuro-Ophthalmology: The Role for Evidence

Neuro-ophthalmic diseases affect relatively small numbers of patients. Thus, there are only a few good-quality clinical trials for their treatments (1-3). Given these facts, neuro-ophthalmologists faced with an ailing patient believe that they have to 'do something.' They often choose therapies based on evidence gathered from underpowered or poorly conducted clinical trials, anecdotal case reports, and even data from animal models or cell culture. We recently wrote an editorial in the Journal of Neuro-Ophthalmology describing a carefully designed, ongoing trial using an antiapoptotic agent in the treatment of patients with nonarteritic anterior ischemic optic neuropathy (NAION) (4). Concern has been raised about withholding systemic corticosteroids in these patients. Here, we address the ethical question of whether a physician must offer an unproven therapy to a patient with an otherwise untreatable disease.