Report from the national eye institute workshop on neuro-ophthalmic disease clinical trial endpoints: Optic neuropathies

Leonard A. Levin, Mohor Sengupta, Laura J. Balcer, Mark J. Kupersmith, Neil R. Miller

Research output: Contribution to journalArticlepeer-review

Abstract

This all-day meeting covered a large number of topics in depth. The following summarizes some of the main conclusions relevant to carrying out clinical trials related to various optic neuropathies. Papilledema (e.g., idiopathic intracranial hypertension): Because visual acuity and color vision are not affected until severe damage has occurred, mean deviation or other measures of progression on automated perimetry are optimal outcome measures for monitoring the effects of papilledema on the visual system. Leber hereditary optic neuropathy: Because of the profound loss of central vision in this condition, assessments of the central visual field are not helpful in determining treatment effects. Although the use electrophysiologic measures can be considered, optimal primary outcome measures have yet to be established. Dominant optic atrophy: The Low Vision Cambridge Color Test can be used in patients with visual acuity >20/800 and has been found to be useful in assessing color discrimination. Optic neuritis: Given that the visual acuity in patients with optic neuritis improves spontaneously over time, clinical measurements of visual function such as central acuity, color vision, or visual field can be less helpful. Electrophysiologic testing—specifically, the P100 latency of the visual evoked potential—may provide evidence for remyelination, and optical coherence tomography of the retinal ganglion cell/inner plexiform layer and peripapillary retinal nerve fiber layer can serve as secondary outcome measures. Glaucoma: The results of automated perimetry—with clustering the timing of visual field examinations in order to increase the ability to detect change—can be used as primary outcome measures. Measurements of contrast sensitivity, color vision, and visual acuity can be used but may be less sensitive or specific. There is no consensus on structural endpoints, in part because the degree of correlation with clinically meaningful functional changes is not yet sufficient. Clinical trial issues applicable to multiple optic neuropathies: A variety of trial designs can be used for rare optic neuropathies, in which the number of participants is likely to be small. Similarly, diseases in which there is severe visual loss can benefit from outcome measures that include patient quality of life. A variety of structural measures continue to be developed, which may eventually serve as primary outcomes once there is evidence for sufficient strength of the association with a clinically meaningful outcome.

Original languageEnglish (US)
Article number30
JournalInvestigative Ophthalmology and Visual Science
Volume62
Issue number14
DOIs
StatePublished - Nov 2021

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems
  • Cellular and Molecular Neuroscience

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