视网膜母细胞瘤的单药与三药动脉内化疗。

IF 4.1 1区医学 Q1 OPHTHALMOLOGY

American Journal of Ophthalmology Pub Date : 2024-10-10 DOI:10.1016/j.ajo.2024.09.037

NAJAH O. ALSHAHRANI , ABEER ALDHAWI , ZHAO XUN FENG , KELVIN CHAU , ASHWIN MALLIPATNA , PRAKASH MUTHUSAMI , CARMEN PARRA-FARINAS , CHRISTIAN ZAAROUR , FURQAN SHAIKH , BRENDA L. GALLIE , STEPHANIE N. KLETKE

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引用次数: 0

摘要

目的比较单药（美法仑）与三药（美法仑、拓扑替康、卡铂）动脉内化疗（IAC）用于视网膜母细胞瘤（RB）眼部救治的眼部和全身预后。方法回顾2016年至2024年间接受过一次或多次IAC手术且随访至少6个月的小于18岁的RB患儿。数据包括临床特征、IAC手术细节、额外的救眼治疗、并发症和随访。主要结果包括 IAC 眼部和全身并发症、眼内复发、眼外扩展、转移和死亡。次要结果包括肿瘤反应、眼部存活率和无复发眼部存活率。对单药组与三药组进行了比较分析。结果对 37 名儿童（24 名单侧 RB）的 38 只眼睛进行了复查。有两只眼睛（2 名儿童）在接受单药 IAC 治疗后又接受了多药 IAC 治疗，因此被排除在外。在纳入的 35 名患儿中，1 名患儿使用了双侧三剂 IAC。IAC（中位数，3次；范围，1-4次）被用作主要治疗（21眼）或辅助治疗（15眼）。13只眼睛采用单药化疗，23只眼睛采用三药化疗。IAC 后，25 只眼睛需要额外的救眼治疗（69% 单药对 70% 三药，P=0.983）。在最终随访中，三药组更有可能获得非常好的部分或完全肿瘤反应（91% 对 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）。眼球挽救率为 72%。两年眼部存活率为 72.2% (95% CI 57.2-87.2) ，三药组的存活率更高（82.6% 对 53.8%；P=0.059）。单药组和三药组分别有31%和52%的眼睛出现眼部并发症（P=0.215）。单药组和三药组的全身并发症发生率分别为 38% 对 74%（P=0.036）。中位随访 34.2 个月（14.5-87.0 个月），未观察到眼外扩展、转移或死亡。结论：与单药相比，三药 IAC 与 RB 肿瘤反应和眼部存活率的改善相关，但无复发眼部存活率相似。虽然三联药剂 IAC 的并发症较多，但大多数是轻微或一过性的。

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Single- Versus Triple-Agent Intra-Arterial Chemotherapy for Retinoblastoma

Purpose

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.

Design

Retrospective single-institutional clinical cohort study.

Methods

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^rb plot graphically illustrated additional treatments following IAC.

Results

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.

Conclusions

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.

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来源期刊

American Journal of Ophthalmology 医学-眼科学

CiteScore

9.20

自引率

7.10%

发文量

406

审稿时长

36 days

期刊介绍： The American Journal of Ophthalmology is a peer-reviewed, scientific publication that welcomes the submission of original, previously unpublished manuscripts directed to ophthalmologists and visual science specialists describing clinical investigations, clinical observations, and clinically relevant laboratory investigations. Published monthly since 1884, the full text of the American Journal of Ophthalmology and supplementary material are also presented online at www.AJO.com and on ScienceDirect. The American Journal of Ophthalmology publishes Full-Length Articles, Perspectives, Editorials, Correspondences, Books Reports and Announcements. Brief Reports and Case Reports are no longer published. We recommend submitting Brief Reports and Case Reports to our companion publication, the American Journal of Ophthalmology Case Reports. Manuscripts are accepted with the understanding that they have not been and will not be published elsewhere substantially in any format, and that there are no ethical problems with the content or data collection. Authors may be requested to produce the data upon which the manuscript is based and to answer expeditiously any questions about the manuscript or its authors.