Prospective exploratory study to assess the safety and efficacy of aflibercept in cystoid macular oedema associated with retinitis pigmentosa

Abstract

Aims: To report the safety and efficacy of intravitreal aflibercept (Eylea) (ivA) for retinitis pigmentosa-associated cystoid macular oedema (RP-CMO) at 12 months via mean central macular thickness (CMT) and reported adverse events.

Methods: A prospective, exploratory, phase II, non-randomised, single-centre, open-label, 1-arm clinical trial involving 30 eyes of 30 patients. Serial ivA was given via loading dose (three injections) followed by treat and extend protocol over 12 months.

Results: Twenty-nine out of 30 (96.7%) patients completed 12 months of follow-up. A total of four to 11 injections per patient were given over the 12 month study. No statistically significant reduction of CMT or visual acuity (VA) improvement was demonstrated in the group overall. Eleven out of 29 (37.9%) participants were considered as 'responders', demonstrating at least an 11% reduction of CMT at 12 months on spectral domain optical coherence tomography compared with baseline. A reduction of CMT by mean (SD) 28.1% (12.9 %) was observed in responders at 12 months, however, no statistically significant corresponding improvement in best corrected VA was seen. Baseline characteristics were similar between responder and non-responder groups. No clinically significant adverse events were deemed secondary to ivA.

Conclusion: This first prospective exploratory study demonstrates both the safety and acceptability of serial ivA in patients with RP-CMO, effective at reducing CMT in 37.9% of patients. All patients demonstrating anatomical response did so after their first injection. Longer duration of CMO did not negatively affect response to anti-VEGF. Further study in a larger cohort of patients with shorter CMO duration would be valuable to better establish the utility of VEGF blockade in RP-CMO.

Trial registration numbers: EudraCT (2015-003723-65); ClinicalTrials.gov (NCT02661711).

Keywords: macula; retina.

Figure 1

A graph demonstrating mean change in central macular thickness from baseline to 6 months post-baseline, and baseline to 12 months post-baseline in responders only (n=11).

Figure 2

Two representative examples of responders: (A) and (C) show spectral domain optical coherence tomography (SDOCT) baseline images of two study participants (study IDs: 04 and 14); (B) and (D) are SDOCT images taken at 1 month post-first aflibercept injection in the same two participants, respectively

Figure 3

A graph demonstrating mean change in central macular thickness from baseline to 6 months post-baseline, and baseline to 12 months post-baseline in responders only (n=11).