Real-World Applicability of Anti-VEGF Trial Criteria for Diabetic Macular Edema.

Abstract

To characterize the real-world eligibility based on systemic medical criteria of patients receiving anti-VEGF injections for DME, including potential rates of trial exclusion and associated patient demographics. Cross-sectional study. Patients who received at least one anti-VEGF injection for treatment of DME with recorded pre-injection blood pressure (BP) and hemoglobin A1c (A1c) measurements. Data was sourced from United States healthcare organizations enrolled in the TriNetX database. Trial selection criteria were sourced from ClinicalTrials.gov. The primary outcome was the overall rate of trial exclusion across all participants. Secondary outcomes comprised the rate of exclusion based on patient demographics. The most inclusionary ("liberal") criteria allowed for participation with BP ≤180/100, A1c ≤12.0%, no history of myocardial infarction (MI) or cerebrovascular accident (CVA) within 3 months prior to first injection, and no history of end stage renal disease (ESRD). The most exclusionary ("stringent") criteria required BP ≤160/95, A1c ≤10.0%, no history of MI or CVA within 6 months prior to first injection, and no history of ESRD. Application of the liberal and stringent criteria excluded 25.7% and 59.1% of real-world patients, respectively. Compared to White individuals, Black individuals were significantly more likely to be excluded by the liberal criteria (Odds ratio [OR] 1.97; 95% CI, 1.57 to 2.46; P_adj < .001) as well as stringent criteria (OR, 2.29; 95% CI, 1.82 to 2.87; P_adj < .001). Individuals of Other race(s) were significantly more likely to be excluded by the liberal criteria (OR, 1.94; 95% CI, 1.33 to 2.84; P_adj = .004). Systemic medical selection criteria for clinical trials of anti-VEGF therapy for DME exclude a significant portion of real-world patients, with members of racial minority groups being disproportionately affected.