Treatment With Erythropoietin for Patients With Optic Neuritis: Long-term Follow-up

Area of Science:

• Neuroscience
• Ophthalmology
• Clinical Trials

Background:

• Erythropoietin (EPO) is being investigated as a potential neuroprotective agent.
• Optic neuritis is an inflammatory condition affecting the optic nerve, often associated with multiple sclerosis (MS).

Purpose of the Study:

• To assess the long-term safety and efficacy of Erythropoietin (EPO) as an add-on therapy to methylprednisolone in patients with optic neuritis.
• To evaluate the impact of EPO on the conversion rate to multiple sclerosis (MS) in this patient cohort.

Main Methods:

• The TONE trial randomized 108 patients with acute optic neuritis to receive either EPO or placebo alongside methylprednisolone.
• An open-label, 2-year follow-up was conducted after the initial 6-month primary endpoint assessment.

Main Results:

• No significant differences were observed in structural (retinal nerve fiber layer atrophy) or functional (low-contrast letter acuity) visual outcomes between the EPO and placebo groups at 2 years.
• Vision-related quality of life scores were similar across both treatment arms.
• The rate of multiple sclerosis-free survival was higher in the EPO group (53%) compared to the placebo group (38%), but this difference did not reach statistical significance (p=0.068).

Conclusions:

• Erythropoietin (EPO) is well-tolerated as an adjunct to methylprednisolone in acute optic neuritis.
• EPO does not provide significant long-term structural or functional benefits to the visual system in patients with optic neuritis.
• A trend towards reduced MS conversion was observed with EPO, warranting further investigation in larger studies.