Alshahrani Najah O, Aldhawi Abeer, Feng Zhao Xun, Chau Kelvin, Mallipatna Ashwin, Muthusami Prakash, Parra-Farinas Carmen, Zaarour Christian, Shaikh Furqan, Gallie Brenda L, Kletke Stephanie N
From the Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children (N.O.A., K.C., A.M., B.L.G., S.N.K.), Toronto, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto (N.O.A., A.M., B.L.G., S.N.K.), Toronto, Canada; Department of Ophthalmology, Prince Sultan Military Medical City (N.O.A.), Riyadh, Saudi Arabia.
Diagnostic Imaging, Hospital for Sick Children, University of Toronto (A.A., P.M., C.P.-F.), Toronto, Canada.
Am J Ophthalmol. 2025 Jan;269:488-496. doi: 10.1016/j.ajo.2024.09.037. Epub 2024 Oct 10.
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 SWIMMER 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 = .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 = .030). Two-year recurrence-free ocular survival was 63.3% (95% CI 45.7-80.9), similar for both groups (P = .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 = .059). Ocular complications occurred in 31% of eyes in the single- and 52% of eyes in the triple-agent group (P = .215). The rate of systemic complications was 38% versus 74% in the single- versus triple-agent groups, respectively (P = .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.
比较单药（美法仑）与三联药物（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）眼的眼部和全身结局。
回顾性单机构临床队列研究。
对2016年至2024年间接受一次或多次IAC手术且随访至少6个月的18岁以下RB患儿进行回顾。数据包括临床特征、IAC手术细节、额外的保眼治疗、并发症和随访情况。主要结局包括IAC的眼部和全身并发症、眼内复发、眼外扩展、转移和死亡。次要结局为肿瘤反应、眼部存活和无复发生存。对单药组和三联药物组进行比较分析。SWIMMER图以图形方式展示了IAC后的额外治疗。
对37例患儿（24例单侧RB）的38只眼进行了回顾。2只眼（2例患儿）先接受单药IAC，后接受多药IAC，被排除在外。在纳入的35例患儿中，1例接受双侧三联药物IAC。IAC（中位数，3剂；范围，1 - 4剂）被用作主要治疗（n = 21只眼）或次要治疗（n = 15只眼）。13只眼采用单药化疗，23只眼采用三联药物化疗。IAC后，25只眼需要额外的保眼治疗（单药组69% vs三联药物组70%，P = 0.983）。在最终随访时，三联药物组更有可能实现非常好的部分或完全肿瘤反应（91% vs 62%，P = 0.030）。两年无复发生存率为63.3%（95%CI 45.7 - 80.9），两组相似（P = 0.700）。眼球挽救率为72%。两年眼部存活率为72.2%（95%CI 57.2 - 87.2），三联药物组更高（82.6% vs 53.8%；P = 0.059）。单药组31%的眼出现眼部并发症，三联药物组52%的眼出现眼部并发症（P = 0.215）。单药组和三联药物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，未观察到眼外扩展、转移或死亡。
三联药物IAC与RB肿瘤反应改善和眼部存活提高相关，尽管与单药IAC相比无复发生存相似。虽然三联药物IAC的并发症更多，但大多数为轻度或短暂性。
