A Randomized Trial of Levodopa as Treatment for Residual Amblyopia in Older Children

Objective To assess the efficacy and short-term safety of levodopa as adjunctive treatment to patching for amblyopia. Design Randomized, placebo-controlled trial. Participants One hundred thirty-nine children 7 to 12 years of age with residual amblyopia resulting from strabismus, anisometropia, or both combined (visual acuity [VA], 20/50–20/400) after patching. Methods Sixteen weeks of oral levodopa or placebo administered 3 times daily while patching the fellow eye 2 hours daily. Main Outcome Measures Mean change in best-corrected amblyopic-eye VA at 18 weeks. Results At 18 weeks, amblyopic-eye VA improved from randomization by an average of 5.2 letters in the levodopa group and by 3.8 letters in the placebo group (difference adjusted for baseline VA, +1.4 letters; 1-sided P  = 0.06; 2-sided 95% confidence interval, −0.4 to 3.3 letters). No serious adverse effects from levodopa were reported during treatment. Conclusions For children 7 to 12 years of age with residual amblyopia after patching therapy, oral levodopa while continuing to patch 2 hours daily does not produce a clinically or statistically meaningful improvement in VA compared with placebo and patching.