NIGHT study reveals insights into choroideremia progression

Amongst individuals with choroideremia (CHM), focusing on functional and anatomical parameters beyond best-corrected visual acuity (BCVA) could offer more accurate ways to track disease advancement and inform management strategies, potentially leading to improved clinical outcomes for individuals affected by this condition, according to study results.

Conducted as a prospective, observational, multicenter cohort study, the NIGHT study focused on adult males with genetically confirmed CHM, exhibiting visible active disease within the macular region and a baseline BCVA of 34 or more Early Treatment Diabetic Retinopathy Study (ETDRS) letters.

Researchers monitored participants for 20 months, with the primary outcome centered around changes in BCVA at various intervals, with secondary measures, including retinal sensitivity, central ellipsoid zone (EZ) area, and total area of fundus autofluorescence (FAF) up to Month 12.

Overall, 220 patients completed the study. The mean BCVA remained stable over the 20-month period, and a significant proportion of participants exhibited changes in ETDRS letters, underscoring the variability in disease progression among individuals.

The study highlighted the importance of alternative measures such as retinal sensitivity, central EZ area, and total FAF area, which demonstrated greater sensitivity in capturing the subtle nuances of CHM progression compared to BCVA alone. Reductions from baseline to Month 12 were observed across these parameters, indicating their potential as robust indicators of disease advancement.

Reference
MacLaren RE, Lam BL, Fischer MD, et al; NIGHT Study Group. A Prospective, Observational, Non-Interventional Clinical Study of Participants With Choroideremia: The NIGHT Study. Am J Ophthalmol. 2024 Feb 2:S0002-9394(24)00024-2. doi: 10.1016/j.ajo.2024.01.022. Epub ahead of print. PMID: 38311152.