视网膜母细胞瘤伴视神经层后侵犯的辅助化疗：一项全球多中心研究。

IF 5.7 Q1 OPHTHALMOLOGY

Ophthalmology. Retina Pub Date : 2026-05-01 Epub Date: 2025-12-29 DOI:10.1016/j.oret.2025.12.018

Michelle Lin BS , Sarah B. Pike MD , Mark W. Reid PhD , Douglas Chigane MD , Brianne Brown MPH , Swathi Kaliki MD , Jesse L. Berry MD

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引用次数: 0

摘要

目的：评价视网膜母细胞瘤（RB）患者行原发性去核术后视神经膜后侵犯（PLONI）的组织学证据，比较接受辅助化疗和未接受辅助化疗的患者的预后。设计：多中心回顾性队列研究。参与者：来自9个国家的292例RB患者和原发性去核后PLONI的组织病理学证据。方法：采用Fisher精确检验、Welch t检验和比例风险模型（Cox和竞争风险）对接受辅助化疗的患者（n=276）和未接受辅助化疗的患者（n=16）进行比较。对PLONI、大量脉络膜侵犯和孤立PLONI进行亚分析。主要观察指标：眼眶肿瘤复发、转移和死亡。结果：未接受辅助化疗的患者眼眶肿瘤复发率（31%比2%，P < 0.001）、转移率（31%比6%，P = 0.001）和死亡率（38%比9%，P = 0.007）较高。在时间-事件分析中，未接受辅助化疗的患者眼眶肿瘤复发（亚危险比SHR = 19.31, 95% CI: 5.56-67.05）、转移（SHR = 5.39, 95% CI: 1.75-16.60）和死亡（HR = 4.74, 95% CI: 1.75-12.81）的风险更高。PLONI和大量脉络膜侵犯患者的结果相似。在孤立性PLONI患者中，未接受辅助化疗的患者随着时间的推移发生转移的风险明显高于接受辅助化疗的患者（SHR = 40.68, 95% CI: 9.65-171.55, P < 0.001）。所有眼眶肿瘤转移或复发患者均在随访期间死亡（平均持续时间：75.3个月）。结论：对于具有其他高危组织病理特征的RB和PLONI患者，在原发性去核后进行辅助化疗可显著降低眼眶肿瘤的复发、转移和死亡率。尽管与具有多种组织病理特征的患者相比，孤立性PLONI患者经历的事件较少，转移风险较低，但随着时间的推移，辅助化疗仍然降低了转移风险。

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Adjuvant Chemotherapy in Retinoblastoma with Postlaminar Optic Nerve Invasion

Purpose

To evaluate outcomes of patients with retinoblastoma (RB) who underwent primary enucleation with histopathologic evidence of postlaminar optic nerve invasion (PLONI), comparing those who received adjuvant chemotherapy to those who did not.

Design

Multicenter retrospective cohort study.

Participants

A total of 292 patients across 9 countries with RB and histopathologic evidence of PLONI after primary enucleation.

Methods

Patients who received adjuvant chemotherapy (n = 276) were compared with those who did not (n = 16) using Fisher exact tests, Welch t tests, and proportional hazard models (Cox and competing risks). Subanalyses were performed for PLONI and massive choroidal invasion and isolated PLONI.

Main Outcome Measures

Orbital tumor recurrence, metastasis, and death.

Results

Patients who did not receive adjuvant chemotherapy had higher rates of orbital tumor recurrence (31% vs 2%, P < 0.001), metastasis (31% vs 6%, P = 0.001), and death (38% vs 9%, P = 0.007). In time-to-event analyses, patients who did not receive adjuvant chemotherapy experienced greater hazard of orbital tumor recurrence (subhazard ratio [SHR] = 19.31, 95% confidence interval [CI]: 5.56–67.05), metastasis (SHR = 5.39, 95% CI: 1.75–16.60), and death (hazard ratio = 4.74, 95% CI: 1.75–12.81). Outcomes were similar among patients with PLONI and massive choroidal invasion. Among patients with isolated PLONI, those who did not receive adjuvant chemotherapy showed significantly greater hazard of metastasis over time than those who received adjuvant chemotherapy (SHR = 40.68, 95% CI: 9.65–171.55, P < 0.001). All patients with metastasis or orbital tumor recurrence eventually died during follow-up (mean duration: 75.3 months).

Conclusions

In patients with RB and PLONI with additional high-risk histopathologic features, initiating adjuvant chemotherapy after primary enucleation significantly reduced rates of orbital tumor recurrence, metastasis, and death. Although patients with isolated PLONI experienced fewer events and had a lower metastatic risk compared with those with multiple histopathologic features, adjuvant chemotherapy still reduced metastatic risk over time.