Personalised versus standardised dosing of occlusion therapy for amblyopia: A randomised controlled trial

Abstract

Objectives

To compare a novel, model-based personalised dosing strategy (PDS) with a standardised (best practice) occlusion dosing strategy (SDS) for the treatment of childhood amblyopia. We have utilised archived clinical trial data sets to develop a statistical model that aims to optimise the prescription of occlusion therapy. This approach, whose effectiveness has not previously been evaluated, incorporates within its underlying model: patient age, severity and type of amblyopia.

Methods

Randomised, parallel group design with PDS and SDS arms each having within or pre-trial optical treatment. Actual worn occlusion for both PDS and SDS groups was objectively recorded using occlusion dose monitors (ODMs).

Results

Of the 94 initially enrolled participants, 50 were male, 44 female, mean [IQR] age 5.53 [4.79–6.02] years. The improvement in logMAR acuity in the 68 participants remaining in the trial subsequent to optical treatment and not having withdrawn did not differ (p = 0.641) between the PDS and SDS groups: median [IQR] improvement 0.250 [0.13−0.39] in the PDS group, 0.225 [0.14−0.32] in the SDS group. A subset (n = 28) of participants were objectively observed to have occluded for every day prescribed. In these, we found that, after allowing for slightly higher prescribed doses of occlusion in the SDS group, adherence in the PDS group to be ∼20% greater than in the SDS group.

Conclusion

Whilst the PDS and SDS groups demonstrated equivalent visual performance and temporal outcomes, there is evidence that the PDS results in improved adherence with therapy likely to have arisen due to increased patient engagement with this novel regimen.