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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).
- retina
- macula
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Footnotes
Contributors SAS, TP, CB, WX, MG, SDE, AK, AW and MM: substantial contributions to the design of the work; acquisition, analysis and interpretation of data for the work; drafting the work; final approval of the version to be published; agreement to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.
Funding This work was supported by grants from the National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital National Health Service Foundation Trust and UCL Institute of Ophthalmology, Fight For Sight (UK), The Macular Society (UK), Moorfields Eye Hospital Special Trustees, Moorfields Eye Charity, by a multiuser equipment grant from The Wellcome Trust [099173/Z/12/Z], the Foundation Fighting Blindness (FFB; USA), Bayer UK, and Retina UK. MM is a recipient of an FFB Career Development Award. CB is part funded/supported by the National Institute for Health Research (NIHR) Biomedical Research Centre based at Guy’s and St Thomas’ NHS Foundation Trust and King’s College London.
Competing interests None declared.
Patient consent for publication Not required.
Ethics approval The protocol of the study adhered to the provisions of the Declaration of Helsinki and was approved by the local ethics committee.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement All data relevant to the study are included in the article or uploaded as supplementary information. Deidentified participant data.