ObjectiveTo compare the clinical efficacy of orthokeratology (ortho-k) and 0.01% atropine for retardation of myopia progression in myopic children. MethodsThis was a retrospective cohort study. A total of 282 patients, aged 8–17 years, were enrolled, including 100 children treated with ortho-k, 84 with 0.01% atropine, and 98 with single-vision spectacles. During the follow-up of 1 year, ortho-k wearers were examined at 1 day, 1 week, 1 month, 3 months after treatment, and thereafter every 3 months, while the others were examined every 3 months by measurements of uncorrected vision, intraocular pressure, refractive power, slit-lamp microscopy, corneal topography, and the lens fitting when necessary. The axial length was measured every 6 months. ResultsPatients with ortho-k had stable uncorrected vision after 1 month of lens wear, all reaching 0 logMAR. The annual axial elongation was 0.23 ± 0.19 mm, 0.22 ± 0.20 mm, and 0.39 ± 0.27 mm in the ortho-k, atropine, and spectacle groups, respectively, with significant difference (F = 23.251, P = 0.000). The axial length was delayed to increase by 41.03% and 43.59% within a year in patients with ortho-k and atropine, respectively, as compared to patients with spectacles (F = 0.006, P = 0.936). The elongation was ≤ 0.3 mm in 69.0% and 66.7% of patients in the two groups, respectively, versus 38.8% in the spectacle group (χ2 = 17.251, P = 0.000). During the follow-up, the rate of corneal staining was 11.0% and 2.0% in the ortho-k and spectacle groups, respectively (χ2 = 8.076, P = 0.003). The use of atropine did not increase corneal staining, but the incidence of related photophobia was 4.8%. No other serious complications were observed. ConclusionOrtho-k lenses and 0.01% atropine can achieve similar efficacy of myopia retardation, which was significantly better than that obtained with single-vision spectacles, in myopic children. The risk of corneal staining after ortho-k wear may be slightly higher than that with spectacles, but could be well controlled.