The Spectrum of Functional, Structural, and Patient-Reported Outcomes in Intermediate Age-Related Macular...

Area of Science:

• Ophthalmology
• Clinical Research
• Biomedical Science

Background:

• Intermediate age-related macular degeneration (iAMD) presents an unmet medical need for effective therapies.
• The MACUSTAR study is a prospective European multicenter cohort study designed to validate endpoints for iAMD.
• Characterizing iAMD requires a multidimensional approach beyond current capabilities.

Purpose of the Study:

• To describe the heterogeneity of assessments in the iAMD baseline cohort of the MACUSTAR study.
• To validate structural, functional, and patient-reported iAMD endpoints for future clinical trials.
• To provide a comprehensive characterization of iAMD through a wide range of assessments.

Main Methods:

• Administered a wide range of structural (e.g., OCT, fundus photography) and functional (e.g., visual acuity, microperimetry) assessments across 20 European sites.
• Incorporated patient-reported outcomes using the Vision Impairment in Low Luminance (VILL) questionnaire.
• Investigated associations between variables using Phi coefficients, Pearson correlation, and age-corrected regression models.

Main Results:

• Included 585 individuals with iAMD (mean age 72 ± 7 years; 66% women).
• Observed varying degrees of structural changes, including pigmentary abnormalities, reticular pseudodrusen, and retinal pigment epithelium atrophy.
• Reported mean best-corrected visual acuity of 0.03 logMAR, low-luminance visual acuity of 0.24 logMAR, and VILL subscale scores of 2 ± 2 to 2 ± 3 logits.

Conclusions:

• The broad spectrum of assessments indicates that iAMD is a diverse and heterogeneous disease.
• Further in-depth characterization of iAMD is necessary to fully understand its complexities.
• Enhanced characterization will facilitate novel enrichment strategies for iAMD clinical trials.