Equivalent effectiveness of a prescription binocular treatment for amblyopia in real-world practice.

Abstract

Gaps often exist between a therapy's performance in a controlled research setting and outcomes in routine clinical practice. A prescription binocular treatment delivered via head-mounted display has demonstrated efficacy for improving visual acuity in children with amblyopia in a pivotal randomized controlled trial (RCT). A retrospective patient registry has been established to collect real-world data on the treatment. To evaluate the real-world effectiveness of this treatment by benchmarking outcomes from a patient registry against those from the RCT. A retrospective analysis of a multicenter US patient registry was completed. The Registry was queried for patients who matched key eligibility criteria of the RCT with respect to age, amblyopia type and severity, and prior treatment exposure. The primary outcome was the change in amblyopic eye best-corrected visual acuity from baseline to the 12-week follow-up visit. These outcomes were compared with the historical intent-to-treat cohort (n = 45) from the RCT using a two one-sided tests procedure for equivalence with an equivalence margin of ±0.75 lines. Among the 40 registry patients meeting query criteria, mean age was 5.7 ± 1.0 years, and 21/40 (53%) had a history of prior treatment. Amblyopic eye best-corrected visual acuity improved a mean 1.7 lines (95% CI = 1.2-2.2), consistent with statistical equivalence within the prespecified ±0.75-line margin compared with RCT participants (1.8 lines; 95% CI = 1.4-2.3; two one-sided tests <i>P</i> = 0.038). No adverse safety events were reported. Patients prescribed this treatment for amblyopia in clinical practice achieved visual acuity improvements consistent with those observed in the RCT, indicating that effectiveness generalizes to real-world settings.