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

Abstract

AimsTo 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.MethodsA 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.ResultsTwenty-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.ConclusionThis 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 numbersEudraCT (2015-003723-65); ClinicalTrials.gov (NCT02661711).

Management of Cystoid Macular Edema in Retinitis Pigmentosa: A Systematic Review and Meta-Analysis

“…However, in a recent study approaching the efficacy of intravitreal aflibercept (2 mg) with a 3+TAE protocol, the treatment failed to achieve a significant overall reduction of CMT, although all the 30 eyes responded after the 1st injection. The responder rate was 37.9% in this study ( 20 ).…”

“…On the other hand, in the 7 patients treated with oral minocycline (100 mg bid for 12 m), 15 adverse events (3 ocular and 12 non-ocular) were recorded within 16 months after the beginning of treatment (NCT02140164). No adverse effects were reported in anti-VEGF therapy, LASER therapy, PPV, ketorolac eyedrops and lutein supplement for the treatment of RP-CME ( 3 , 20 , 21 , 25 , 32 , 33 , 40 , 41 ).…”

“…In the meantime, intravitreal bevacizumab (1.25 mg) was reported to improve VA from 5/400–20/100 to 20/200–20/63 in 13 treated eyes ( 33 ). However, intravitreal aflibercept (2 mg) administrated with a 3+TAE protocol failed to show any help in BCVA ( 20 ).…”

“…For IVTA ( 27 ), subtenon TA ( 10 ) and intravitreal dexamethasone implant treatments ( 11 , 16 , 19 ), multiple injections were needed because of recurrence of CME. Repeated injections were also common in anti-VEGF therapy for RP-CME ( 20 , 33 ).…”

“…Two systematic reviews and one meta-analysis have been published regarding the treatment of RP-CME, with results of studies up to 2016 summarized and analyzed ( 4 , 8 , 12 ). Nevertheless, during the past 5 years, more evidence has been published on the application of CAIs ( 13 – 18 ), steroids ( 10 , 11 , 13 , 14 , 16 , 19 ), anti-VEGF therapy ( 20 ), and LASER treatment ( 21 ). Therefore, we conducted this updated systematic review and meta-analysis, to summarize the existing evidence on the treatment of RP-CME.…”