Pharmacogenomic study on anti-VEGF medicine in treatment of macular Neovascular diseases: a study protocol for a prospective observational study

BACKGROUND: Macular neovascular diseases can cause severe vision loss. A newly approved anti—VEGF drug Conbercept has shown good efficacy and safety in rigorous random controlled trials (RCT), however, it cannot fully reflect the clinical application of Conbercept in real world clinical practice. Moreover, anti-VEGF drugs are expensive and often require multiple treatments, and some patients have poor or even no response to the drugs,this resulted enormous waste of medical resources. Therefore, how to find out those patients who have good response, and how to develop individualized therapeutic regimen in real world need to be urgently investigated in the aspect of pharmacogenomics and pharmacometabolomics. METHODS: This study is a multicenter, prospective, observational study of Conbecept treating macular neovascular diseases in China. Patients suffered from age-related macular degeneration, polypoidal choroidal vasculopathy, and pathological myopia who already planned to receive Conbercept treatment will be recruited. We aimed to enroll more than 5000 patients from 43 ophthalmic centers in China. Patients’ clinical data and blood samples will be collected during the one-year follow-up period. Finally, the safety and efficacy of Conbercept, and the potential predictors of patients’ response to Conbercept will be investigated by pharmacogenomics and pharmacometabolomics analysis. DISCUSSION: This study will provide important data of Conbercept in treating macular neovascular diseases in real world. Besides, finding the predictor of patients’ response will help doctor make more precise individualized therapeutic regimens. TRIAL REGISTRATION: ClinicalTrials.gov, NCT03128463. Registered on 9 March 2017. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s12886-018-0812-4) contains supplementary material, which is available to authorized users.