Efficacy and safety of minocycline in retinitis pigmentosa:a prospective,open-label,single-arm trial  

作　　者：Yuxi Chen Yuan Pan Yanyan Xie Yuxun Shi Yao Lu Yiwen Xia Wenru Su Xiaoqing Chen Zuoyi Li Minzhen Wang Siyu Miao Yating Yang Chenjin Jin Guangwei Luo Shixian Long Hui Xiao Chuangxin Huang Jian Zhang Dan Liang 

机构地区：[1]Department of Ocular Immunology,State Key Laboratory of Ophthalmology,Zhongshan Ophthalmic Center,Sun Yat-sen University,Guangdong Provincial Key Laboratory of Ophthalmology and Visual Science,Guangdong Provincial Clinical Research Center for Ocular Diseases,Guangzhou,China

出　　处：《Signal Transduction and Targeted Therapy》2025年第1期271-277,共7页信号转导与靶向治疗(英文)

基　　金：supported by the Sun Yat-sen University Clinical Research 5010 Program(2018012).

摘　　要：Retinitis pigmentosa(RP)is characterized by progressive photoreceptor cells death accelerated by the proliferation and activation of microglia pathologically.No consensus exists on the treatment.Minocycline is recognized as a microglia inhibitor with great anti-inflammatory and neuro-protective functions.However,efficacy of minocycline in RP patients is lacking.We conducted a prospective,open-label,and single-arm trial,in which daily oral minocycline of 100 mg was administered for 12 months in RP patients with light-adapted 30 Hzflicker electroretinography(ERG)amplitude>0µV in at least one eye(NCT04068207).The primary outcome was the proportion of participants with improvement in the ERG amplitude at month 12.The secondary outcomes included improvements of the following items:other ERGs amplitudes,visualfield,best-corrected visual acuity,contrast sensitivity,color vision,and NEI-VFQ-25.35 of 288 patients met inclusive criteria were enrolled(median[IQR]age,36[31-45]years;17 female[48.6%]).32 participants completed all examinations,while 3 participants completed the 12-month online visit via conducting NEIVFQ-25.The primary outcome showed improvement was 34.3%(12 of 35[95%CI 19.1-52.2]).Similarly,all secondary outcomes showed improvements.Adverse events were reported in 22 participants(62.9%)and were all resolved without extra medication during the study period.No severe adverse events were recorded.Ourfindings identified daily oral minocycline of 100 mg for 12 months was beneficial in improving the visual function of RP patients with good safety.This study indicates minocycline may be a promising therapy for RP,but a randomized controlled trial is still needed of further exploration.

关 键 词：protective PROSPECTIVE adapted 

分 类 号：R73[医药卫生—肿瘤]