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Durable recovery from amblyopia with donepezil.

Carolyn Wu1,2, Eric D Gaier3,4,5, Bharti R Nihalani3,4

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Donepezil, an acetylcholinesterase inhibitor (AChEI), improved vision in older children and adults with residual amblyopia. This treatment enhanced neuroplasticity, suggesting pharmacological manipulation can overcome critical windows for visual recovery.

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Area of Science:

  • Ophthalmology
  • Neuroscience
  • Pharmacology

Background:

  • Elevated neuroplasticity thresholds limit visual recovery in older amblyopia patients.
  • Acetylcholinesterase inhibitors (AChEI) show potential for enhancing visual neuroplasticity and treating amblyopia in animal models.

Purpose of the Study:

  • To investigate if donepezil, a common AChEI, can restore visual function in older children and adults with residual amblyopia.
  • To assess the safety and efficacy of donepezil in treating amblyopia beyond the typical critical period.

Main Methods:

  • An open-label pilot study treated 16 participants (ages 9-37) with residual amblyopia using daily oral donepezil for 12 weeks.
  • Dosage was adjusted based on age and visual acuity response, with patching for participants under 18.
  • Visual acuity was measured at 22 weeks, 10 weeks post-treatment cessation.

Main Results:

  • Mean visual acuity in the amblyopic eye improved by 1.2 lines.
  • 25% of participants (4/16) achieved a ≥2 line improvement.
  • Visual gains were maintained 10 weeks after discontinuing donepezil and were consistent across age groups.

Conclusions:

  • Donepezil treatment led to significant visual improvements in older children and adults with residual amblyopia.
  • Findings support the potential for pharmacological interventions to enhance visual cortical plasticity and treat amblyopia.
  • Further placebo-controlled trials are warranted to confirm these preliminary results.