Real-world Effectiveness of 0.01% Atropine for Primary Prevention of Myopia in Children: A Retrospective Cohort Study

Abstract Purpose: The efficacy of atropine 0.01% for preventing myopia onset remains controversial despite its widespread use. Its effectiveness in preventing incident myopia was evaluated in a real-world clinical setting. Methods: This retrospective cohort study included 290 non-myopic or pre-myopic children (157 receiving atropine 0.01% and 133 untreated controls) attending a tertiary eye center between January 2018 and October 2024. Longitudinal changes in spherical equivalent (SE) were analyzed using linear mixed-effects models. Time to myopia onset (SE ≤ −0.50 D) was evaluated using Kaplan–Meier analysis and Cox proportional hazards regression. Multiple imputation and overlap-weighting analyses were performed as sensitivity analyses. Results: Myopia developed in 96 of 157 children (61.1%) receiving atropine and 98 of 133 controls (73.7%). In the fully adjusted model, the group-by-time interaction was not significant (β = 0.021, 95% CI −0.046 to 0.089; P = 0.536), indicating similar rates of refractive progression. Baseline SE was the strongest predictor (β = 0.888, P < 0.001). Kaplan–Meier analysis showed no significant difference (P = 0.182). Cox regression yielded a hazard ratio of 0.76 (95% CI 0.57–1.00; P = 0.052), while overlap weighting attenuated the association (HR 0.89, 95% CI 0.65–1.20; P = 0.44). Conclusion: In this real-world cohort, atropine 0.01% was not associated with a significant reduction in myopia onset or refractive progression. These findings do not support routine use of atropine 0.01% solely for preventing myopia onset.