A 4-year longitudinal study of 555 patients treated with ranibizumab for neovascular age-related macular degeneration.

Objective: To investigate the visual outcome, pattern of discontinuation, ocular complications, and mortality of patients treated with a variable ranibizumab dosing regimen for neovascular age-related macular degeneration (AMD) for 4 years.

Design: Retrospective chart review supplemented with clinical examination.

Participants: Six hundred eyes of 555 patients initiated intravitreal treatment with vascular endothelial growth factor inhibition for neovascular AMD in 2007 in a community-based hospital.

Methods: Patient data from a database were retrieved from 2007 through 2011. Descriptive evaluation of the main outcome measures was carried out for the cohort of patients. A group of patients who had been discontinued because of apparent disease inactivity was reexamined.

Main Outcome Measures: Best-corrected visual acuity (BCVA; Snellen), number of intravitreal injections, causes of discontinuations, ocular complications, and standardized mortality rate.

Results: One hundred ninety-two eyes (32%) were still receiving active treatment after 4 years. The mean BCVA in the 192 eyes was unchanged from the start (baseline, 0.30; 4-year follow-up, 0.32; P>0.3). Visual acuity after the third loading dose was associated significantly with the outcome (P<0.0001) and was a better predictor than baseline acuity. The mean number of injections was 5.5 per year. For 408 eyes (68%), discontinuation of treatment was motivated by the following 4 reasons: lack of apparent treatment response (28%), failure to appear at follow-up (11%), death (9%), and disease inactivity (20%, 120 eyes). Treatment was resumed later in 18% of patients discontinued because of inactivity. Sixty-seven eyes were reexamined in 2012 from the group of patients with disease inactivity. The final visual acuity by then had decreased significantly from the time of discontinuation, from 0.38 to 0.15 (P = 0.001). Endophthalmitis occurred in 2 eyes of 7584 injections. A total of 125 patients had died, corresponding to 75% of the mean mortality in the community.

Conclusions: One third of the eyes were still receiving active treatment after 4 years and had stable visual acuity. One third of fellow eyes (eyes at risk) started treatment during the 4 years. One fifth of discontinued eyes resumed treatment, indicating that close follow-up should be maintained for patients discontinued because of disease inactivity. The ocular complication rate was 0.2%, and the mortality rate was below expected.