Anti-VEGF Treatment in Pseudoxanthoma Elasticum – Risk factors and Visual Outcome

Abstract

Purpose : To assess the prevalence and onset of anti-VEGF treatment in patients with Pseudoxanthoma elasticum (PXE), the treatment course and outcome.

Methods : This retrospective study included patients from adedicated PXE clinic at the Department of Ophthalmology, University of Bonn, Germany. Inclusion criteria were the genetically confirmed diagnosis of PXE and an observation time of ≥ 12 months. Best-corrected visual acuity (BCVA) at baseline, age at anti-VEGF-therapy initiation, and BCVA drop events at exudation onset (worsening of BCVA of 2 or more lines) were documented. Further, we assessed the number of injections during the first year, time to treatment of the second eye, total number of injections, and BCVA over time.

Results : A total of 106 eyes of 53 patients were analyzed during a median observation period of 77 months (IQR 49 – 126), with a median number of 28.0 injections (IQR 9.8 – 43.5).injections per patient During the observation period, 8 patients received no injections in both eyes (median age at baseline 38.1 years). 11 patients underwent anti-VEGF treatment in one eye (median age 47.2 years), and 34 patients in both eyes (median age 51.8 years). The median age at the first anti-VEGF treatment was 52.80 years (IQR 47.2 – 57.6). Once the first eye was treated, the median time to fellow eye treatment was 9.6 months (IQR 2.1 – 32.4, range 0 – 122) and was not significantly associated with sex or age. The median number of injections was 5.5 per eye in the first year (IQR 3 – 7) and was highly associated with the total number of injections in the observation period (2.27, CI 1.33 – 3.21, p<0.001). BCVA at the last follow-up visit was associated with baseline VA (0.63, 0.37 – 0.90, <0.001, R²=0.318), and also with a VA drop at onset of exudation (-0.33, -0.69 – 0.03, p=0.035, R²=0.339).

Conclusions : Anti-VEGF treatment is required for most PXE patients at a relatively young age. Once treatment in one eye is initiated, the time interval to fellow eye treatment is relatively short. A BCVA drop at onset of exudation is a risk factor for worse visual outcomes, indicating that a treatment should be initiated early and before exudation affects the central retina. This group of patients might particularly benefit from longer-acting anti-VEGF therapeutics and delivery systems given the young age of onset and intensive treatment needs.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.