Department of Radiology, the First Affiliated Hospital, Jinan University, No.613, Huangpu West Road, Tianhe District, Guangzhou, Guangdong, 510627, People's Republic of China.
Graduate College, Jinan University, No.613, Huangpu West Road, Tianhe District, Guangzhou, Guangdong, 510627, People's Republic of China.
BMC Cancer. 2019 Jul 15;19(1):693. doi: 10.1186/s12885-019-5844-5.
To evaluate the safety and efficacy of intra-arterial chemotherapy (IAC) for the primary or secondary treatment of infants diagnosed with advanced retinoblastoma before 3 months of age.
This single-center retrospective study included 39 infants (42 eyes) aged ≤3 months who were diagnosed with unilateral or bilateral advanced intraocular retinoblastoma (group D and E eyes) and received IAC as primary or secondary treatment between June 2012 and February 2017. Based on each patient's therapeutic history and response to chemotherapeutic drugs, melphalan, topotecan, and/or carboplatin were used for IAC. The main outcomes included the technical success rate for IAC, survival rates, and adverse events.
In total, 29 and 13 eyes received IAC as primary and secondary treatments, respectively. Catheterization was successful in 136 of 137 procedures. All eyes in the secondary IAC group had previously received intravenous chemotherapy. The mean number of IAC sessions for each eye was 3 (range, 2-6). The 2-year ocular survival rates were 80.7% (95% confidence interval [CI], 58.9-91.7) in the primary IAC group and 91.7% (95% CI, 53.9-98.8) in the secondary IAC group. During the follow-up period, 1 patient with unilateral disease (group E) developed extraocular disease and died. The 2-year recurrence-free survival rates in the primary and secondary IAC groups were 71.9% (95% CI, 49.4-85.7) and 75.0% (95% CI, 40.8-91.2), respectively. During each catheterization procedure, the main complications included eyelid erythema (2.4%), fundus hemorrhage (11.9%), myelosuppression (7.7%), transient vomiting and hair loss (2.6%), and transient pancytopenia (2.6%). Prolonged complications included phthisis bulbi (19.0%), vision loss (19.0%), poor vision (9.5%), and cataract (2.4%). There was no case of stroke, neurological impairment, secondary malignant tumor, or metastasis.
Our findings suggest that IAC, whether primary or secondary, is effective and fairly safe for the management of advanced retinoblastoma in infants aged < 3 months. However, adverse events related to intra-arterial injection and the visual outcomes cannot be neglected and require further investigation.
评估在 3 个月龄以下被诊断患有晚期视网膜母细胞瘤的婴儿进行经动脉化疗（IAC）作为一线或二线治疗的安全性和疗效。
本单中心回顾性研究纳入了 2012 年 6 月至 2017 年 2 月期间，接受 IAC 作为一线或二线治疗的 39 名（42 只眼）≤3 个月龄的单侧或双侧晚期眼内视网膜母细胞瘤（D 组和 E 组眼）患儿。根据每位患者的治疗史和对化疗药物的反应，使用氨甲蝶呤、拓扑替康和/或卡铂进行 IAC。主要结局包括 IAC 的技术成功率、生存率和不良事件。
共有 29 只眼接受了 IAC 作为一线治疗，13 只眼作为二线治疗。在 137 次介入中，136 次导管插入术成功。二线 IAC 组所有眼均曾接受过静脉化疗。每只眼接受 IAC 治疗的平均次数为 3 次（范围：2-6 次）。一线 IAC 组的 2 年眼存活率为 80.7%（95%置信区间 [CI]，58.9-91.7），二线 IAC 组为 91.7%（95% CI，53.9-98.8）。在随访期间，1 名单侧疾病（E 组）患儿发生眼外疾病并死亡。一线和二线 IAC 组的 2 年无复发生存率分别为 71.9%（95% CI，49.4-85.7）和 75.0%（95% CI，40.8-91.2）。每次导管插入术的主要并发症包括眼睑红斑（2.4%）、眼底出血（11.9%）、骨髓抑制（7.7%）、短暂呕吐和脱发（2.6%）和短暂全血细胞减少症（2.6%）。长期并发症包括眼球萎缩（19.0%）、视力丧失（19.0%）、视力不佳（9.5%）和白内障（2.4%）。无中风、神经功能障碍、继发恶性肿瘤或转移的病例。
我们的研究结果表明，IAC 作为一线或二线治疗方案，对于<3 个月龄的晚期视网膜母细胞瘤婴儿是有效且相对安全的。然而，与动脉内注射相关的不良事件和视觉结局不容忽视，需要进一步研究。
