视网膜母细胞瘤的动脉内化疗：357只眼的疗效分析——巴西一家转诊中心的13年经验

Teixeira Luiz F, Macedo Carla R P D, Fonseca José R F, Morales Bruna, Mangeon Monique K, Miranda Bruno A, Casaroli-Marano Ricardo, Sallum Juliana M F

Department of Ophthalmology, Federal University of São Paulo (UNIFESP), São Paulo, Brazil; Pediatric Oncology Institute - GRAACC, Federal University of São Paulo, São Paulo, Brazil.

Pediatric Oncology Institute - GRAACC, Federal University of São Paulo, São Paulo, Brazil.

Ophthalmol Retina. 2025 Aug;9(8):798-806. doi: 10.1016/j.oret.2025.02.013. Epub 2025 Feb 17.

OBJECTIVE

To evaluate the outcomes of intra-arterial chemotherapy (IAC) for the treatment of naive and nonnaive retinoblastoma eyes. Ocular survival rates, risk factors for enucleation, ocular complications, metastatic disease, and overall survival were analyzed.

DESIGN

A retrospective, single-institution study.

PARTICIPANTS

A total of 300 patients treated with IAC between April 2010 and April 2023 were included.

INTERVENTIONS

During IAC infusions, 1 to 3 drugs were used (melphalan, 3.0-7.5 mg; topotecan, 0.3-2.0 mg; and carboplatin, 20-50 mg). Adjuvant therapy was used as needed to consolidate treatment.

MAIN OUTCOME MEASURES

Ocular survival rates, ocular complications, and the risk factors for enucleation were measured.

RESULTS

A total of 357 eyes were treated with 1536 IAC infusions, with a median of 4 cycles per eye, and followed for 60.69 months. The Kaplan-Meier estimates for the overall ocular survival were 90% at 1 year, 89% at 2 years, and 86% at 5 years. No difference in ocular survival was found between IAC indications (primary 88% vs. secondary 85% vs. bridge 89%; P = 0.52) and the use of tandem therapy (tandem 85% vs. no tandem 87%; P = 0.93). Intravitreal chemotherapy was used as adjuvant therapy in 31.37% and plaque therapy in 5% of the eyes. The group did not receive external beam radiation. Univariable and multivariable analyses showed that the presence of subretinal seeds was significantly associated with an increased risk of enucleation, and the use of ophthalmic artery (OA) ostium in >50% of infusions per eye was a protective factor to avoid enucleation. Retinal and choroidal vascular, ischemic, or atrophic effects were the most frequent complications found in 5.0% of the eyes. Metastatic disease was observed in 0.33% of the patients. The overall 5-year patient survival was 99.3%.

CONCLUSIONS

The use of IAC in different indications (primary, secondary, bridge, and tandem) to treat naive or recurrent-refractory retinoblastomas showed successful results. Most eyes were preserved. Subretinal seeds at presentation were associated with a high enucleation risk. The use of the OA ostium for drug delivery avoided enucleation.

FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

目的

评估动脉内化疗（IAC）治疗初发和非初发视网膜母细胞瘤眼的疗效。分析了眼球生存率、眼球摘除的危险因素、眼部并发症、转移性疾病和总生存率。

设计

一项回顾性单机构研究。

参与者

纳入2010年4月至2023年4月期间接受IAC治疗的300例患者。

干预措施

在IAC输注期间，使用1至3种药物（美法仑，3.0 - 7.5毫克；拓扑替康，0.3 - 2.0毫克；卡铂，20 - 50毫克）。根据需要使用辅助治疗巩固疗效。

主要观察指标

测量眼球生存率、眼部并发症和眼球摘除的危险因素。

结果

共357只眼接受了1536次IAC输注，每只眼的中位数为4个周期，并随访了60.69个月。Kaplan-Meier估计的总体眼球生存率在1年时为90%，2年时为89%，5年时为86%。IAC适应证（初发88% vs. 继发85% vs. 桥接89%；P = 0.52）和串联治疗的使用（串联85% vs. 非串联87%；P = 0.93）之间的眼球生存率无差异。31.37%的眼使用玻璃体腔内化疗作为辅助治疗，5%的眼使用敷贴治疗。该组未接受外照射放疗。单因素和多因素分析显示，视网膜下种植的存在与眼球摘除风险增加显著相关，每只眼超过50%的输注使用眼动脉（OA）开口是避免眼球摘除的保护因素。视网膜和脉络膜血管、缺血或萎缩效应是5.0%的眼中最常见的并发症。0.33%的患者观察到转移性疾病。总体5年患者生存率为99.3%。