Patient selection criteria for pilot studies on amelioration of non-neovascular age-related macular degeneration.

Purposes: Non-neovascular age-related macular degeneration (AMD) is characterized by accumulation of macular drusen, changes in pigmentation of the retinal pigment epithelium, and geographic atrophy. The purposes of this study were to (1) measure the rate of progression of non-neovascular AMD and (2) from the rate data, to propose patient selection criteria for testing drugs to prevent progression of non-neovascular AMD.

Methods: Medical charts were searched for all AMD billing codes, consecutively reviewed, and 51 patients with a median age of 76 years were mined for severity of AMD using a standardized worsening scale from 0 to 6, visual acuity (VA, Snellen), medications or procedures to treat eye diseases, date of eye examinations, age, and sex. Individual eyes, excluding those with cataract, were grouped and compared.

Results: Using all grades, VA (logMAR) was positively correlated with AMD scores (P < 0.0001, n = 66). The median length of time to progress from AMD grade 3 or 4 to the next grade was 1.0 (n = 14) and 1.7 years (n = 7), respectively. Statistical analyses predicted that drug-treated and nontreated groups, each containing 409 grade 3 and 4 AMD eyes, could detect 50% drug inhibition (P = 0.05) in a 2-year trial.

Conclusions: VA measurements and structural AMD grades would be useful markers in clinical trials on non-neovascular AMD. Recruiting only grade 3 and 4 patients may be ideal for time- and cost-efficient pilot drug efficacy studies on moderately progressing non-neovascular AMD.