Real-world Outcomes of Anti-Vascular Endothelial Growth Factor Therapy in Neovascular Age-Related Macular Degeneration in the United States

Abstract

Purpose: Real-world visual outcomes of anti-vascular endothelial growth factor (anti-VEGF) therapy for neovascular age-related macular degeneration (nAMD) have been reported in cohorts outside of the United States. This study sought to assess the relationship between presenting visual acuity (VA) and visual outcomes, as well as the potential impact of loss to follow-up, in real-world anti-VEGF-treated nAMD patients from the United States.

Design: Retrospective study of aggregated, longitudinal electronic medical records obtained from a geographically diverse sample of US retina specialists and included in the Vestrum Health Retina Database.

Participants: Inclusion criteria were a diagnosis of nAMD, no previous treatment, and ≥3 monthly anti-VEGF injections in the first 4 months from diagnosis in patients diagnosed between January 2011 and July 2013.

Methods: To model loss to follow-up, mutually exclusive cohorts of nAMD patients with loss to follow-up after specific time points of 6 and 12 months (i.e., no follow-up beyond) were compared with a separate cohort of patients who completed 24 months of follow-up ending prior to July 2015 (n = 2213).

Main outcome measure: VA outcomes were assessed on each cohort as a whole, with additional stratification by baseline VA.

Results: The 6-, 12-, and 24-month cohorts received means of 5.4, 7.3, and 12.1 injections and showed no change, no change, and a mean change of +3.1 letters from baseline (95% confidence interval 1.8-4.4 letters, P < 0.01), respectively. When stratified by baseline VA, nearly all groups lose VA at their respective follow-up periods, except for those with baseline VA of 20/200 or worse.

Conclusions: Real-world nAMD patients in the United States receive fewer anti-VEGF injections and experience worse visual outcomes compared with patients in randomized clinical trials, consistent with non-US studies. Patients with better VA at presentation tend to be particularly vulnerable to vision loss. Compared with other patients, those ultimately lost to follow-up have worse visual outcomes at, or prior to, their final visit, suggesting that loss to follow-up may lead to overestimation of visual outcomes in clinical studies of nAMD.