摘要视网膜母细胞瘤（RB）是致盲、致残、致死的最严重婴幼儿眼病。抑癌基因 RB1缺失导致肿瘤发生。随着治疗方法的不断创新和发展，RB治疗从眼球摘除、外放射治疗、静脉化学药物治疗、到眼动脉介入化学药物治疗，逐步实现了保生命、保眼球和保视力。但是，RB的发生机制仍存在很多空白点，眼内晚期患儿的眼球摘除率居高不下，治疗方法的创新和验证能力有待进一步提高。因此，重视RB发生机制、早期诊断和治疗方法的研究，加强多中心临床试验和转化医学研究，是提高RB整体治疗水平的关键。

abstractsRetinoblastoma (RB) is the most serious eye disease that causes blindness, disability and death in infant. Loss of tumor suppressor gene RB1 leads to tumorigenesis of RB. With continuous innovation and development of new methods, the management of retinoblastoma has experienced a dramatic change from enucleation, external radiotherapy, intravenous chemotherapy, to intra-arterial chemotherapy combined with local treatment, enabling a more favorable outcome for survival, salvage and vision. However, the pathogenesis of RB is unclear in many aspects, the rate of eyeball removal in children with advanced RB remains high, and the innovation of new treatment are needed to be further promoted. Therefore, the key to improve the overall treatment level of RB is to pay attention to the study on pathogenesis, early diagnosis and treatment, as well as strengthen multi-center clinical trials and translational medicine research.