Longitudinal Visual Biomarkers in Dominant Optic Atrophy: A Systematic Review and Meta-Analysis

Abstract

Background: Dominant Optic Atrophy (DOA) causes slowly progressive visual decline usually beginning in childhood. As new therapies come to clinical trial, the choice of biomarkers to be used as clinical trial endpoints has become a critical question to be addressed. Methods: We undertook a systematic review and meta-analysis of studies reporting longitudinal data of any biomarker in DOA patients. Results: In total, seven studies were included in the systematic review, and four presented paired results compatible with meta-analysis. Visual acuity was the only biomarker found with reported longitudinal data. Of the included studies in the meta-analysis, the rate of yearly visual acuity decline (0.022 LogMAR/year., 95% CI: −0.008 to 0.052) was not significantly different from zero (Z = 1.4, p = 0.155). Conclusion: Quantifying this slow rate of visual decline has implications for future study design and suggests that further natural history studies examining alternative biomarkers in DOA are warranted.