Sample size estimation for AQP4-IgG seropositive optic neuritis: Retinal damage detection by optical coherence tomography.

Abstract

We recruited four aquaporin-4 seropositive optic neuritis patients (five eyes) who received glucocorticoid treatment and underwent optical coherence tomography examination. Baseline medians of the macular ganglion cell layer plus inner plexiform layer (mGCIPL) thickness and volume for the eye of interest were 79.67 µm (73.664 ± 18.497 µm) and 0.58 mm3 (0.534 ± 0.134 mm3), respectively. At 2 months, the medians of the mGCIPL thickness and volume were 60.00 µm (51.576 ± 12.611 µm) and 0.44 mm3 (0.376 ± 0.091 mm3), respectively. At 6 months, the medians of the mGCIPL thickness and volume were 59.55 µm (46.288 ± 11.876 µm) and 0.44 mm3 (0.336 ± 0.084 mm3), respectively. Sample size estimate was achieved using two methods based on the mGCIPL thickness and volume data, with five effect sizes considered. The estimate based on the mGCIPL volume showed that 206 patients were needed at the 6-month follow-up; the power was 80% and effect size was 20%. In conclusion, this study detected retinal damage in aquaporin-4 seropositive optic neuritis patients by optical coherence tomography, and estimated the sample size for two-sample parallel designed clinical trials using two methods.