比较单药（美法仑）与三药（美法仑、拓扑替康、卡铂）动脉内化疗 (IAC) 挽救视网膜母细胞瘤 (RB) 眼部的眼部和全身结局。回顾性单机构临床队列研究。<18 岁的儿童对 2016 年至 2024 年间接受一次或多次 IAC 手术并至少进行 6 个月随访的 RB 患者进行了回顾。数据包括临床特征、IAC 程序细节、额外的护眼治疗、并发症和随访。主要结局包括 IAC 的眼部和全身并发症、眼内复发、眼外扩散、转移和死亡。次要结局是肿瘤反应、眼部存活率和无复发眼部存活率。对单药组与三药组进行比较分析。 SWIMMERrb 图以图形方式说明了 IAC 后的额外治疗。对 37 名儿童（24 名单侧 RB）的 38 只眼睛进行了审查。两只眼睛（2 名儿童）接受单剂 IAC，随后接受多剂 IAC，并被排除。在 35 名儿童中，一名接受双侧三药 IAC。 IAC（中位数，3 剂量；范围，1-4）用作主要（n=21 只眼睛）或次要（n=15 只眼睛）治疗。 13只眼采用单药化疗，23只眼采用三药化疗。 IAC 后，25 只眼睛需要额外的眼科治疗（69% 单药治疗，70% 三药治疗，p=0.983）。在最终随访时，三药组更有可能实现非常好的部分或完全肿瘤缓解（91% vs 62%，p=0.030）。两年无复发眼部生存率为 63.3% (95% CI 45.7-80.9)，两组相似 (p=0.700)。全球抢救率为72%。三药组的两年眼部生存率为 72.2%（95% CI 57.2-87.2），较高（82.6% vs 53.8%；p=0.059）。单药组有 31% 的眼睛发生眼部并发症，三药组有 52% 的眼睛发生眼部并发症 (p=0.215)。单药组和三药组的全身并发症发生率分别为 38% 和 74% (p=0.036)。在中位 34.2 个月（范围 14.5-87.0）的随访中，未观察到眼外扩散、转移或死亡。 三联药物 IAC 与改善 RB 肿瘤反应和眼部存活率相关，尽管与单剂相比，无复发眼部存活率相似-代理人。虽然三药 IAC 会出现更多并发症，但大多数都是轻微或短暂的。版权所有 © 2024。由 Elsevier Inc. 出版。

To compare the ocular and systemic outcomes of single- (melphalan) versus triple-agent (melphalan, topotecan, carboplatin) intra-arterial chemotherapy (IAC) for retinoblastoma (RB) eye salvage.Retrospective single-institutional clinical cohort study.Children <18 years with RB who underwent one or more IAC procedures between 2016 and 2024 with minimum 6-month follow-up were reviewed. Data included clinical features, IAC procedural details, additional eye-saving treatments, complications, and follow-up. Primary outcomes included ocular and systemic complications of IAC, intraocular recurrence, extraocular extension, metastasis, and death. Secondary outcomes were tumor response, ocular survival, and recurrence-free ocular survival. Comparative analysis was performed for single- versus triple-agent groups. A SWIMMERrb plot graphically illustrated additional treatments following IAC.Thirty-eight eyes of 37 children (24 unilateral RB) were reviewed. Two eyes (2 children) had single- followed by multi-agent IAC and were excluded. Of 35 included children, one had bilateral triple-agent IAC. IAC (median, 3 doses; range, 1-4) was employed as primary (n=21 eyes) or secondary (n=15 eyes) treatment. Chemotherapy was single-agent in 13 eyes and triple-agent in 23 eyes. Following IAC, 25 eyes required additional eye-saving treatments (69% single- v 70% triple-agent, p=0.983). At final follow-up, the triple-agent group was more likely to achieve very good partial or complete tumor response (91% v 62%, p=0.030). Two-year recurrence-free ocular survival was 63.3% (95% CI 45.7-80.9), similar for both groups (p=0.700). Globe salvage was 72%. Two-year ocular survival was 72.2% (95% CI 57.2-87.2), higher for the triple-agent group (82.6% v 53.8%; p=0.059). Ocular complications occurred in 31% of eyes in the single- and 52% of eyes in the triple-agent group (p=0.215). The rate of systemic complications was 38% v 74% in the single- versus triple-agent groups, respectively (p=0.036). No extraocular extension, metastasis, or death were observed at median 34.2 months (range, 14.5-87.0) follow-up.Triple-agent IAC was associated with improved RB tumor response and ocular survival, though similar recurrence-free ocular survival compared to single-agent. While there were more complications with triple-agent IAC, most were mild or transient.Copyright © 2024. Published by Elsevier Inc.