Thampi Sheila, Hetts Steven W, Cooke Daniel L, Stewart Paul J, Robbins Elizabeth, Banerjee Anuradha, Dubois Steven G, Char Devron, Halbach Van, Matthay Katherine
Department of Pediatrics, University of California, San Francisco School of Medicine, San Francisco, CA, USA.
Clin Ophthalmol. 2013;7:981-9. doi: 10.2147/OPTH.S43398. Epub 2013 May 27.
Intra-arterial administration of melphalan chemotherapy has shown promise in the treatment of retinoblastoma. This report describes our results using superselective intra-arterial melphalan in patients with newly diagnosed retinoblastoma and those who were treated for progression after systemic chemotherapy.
This is a retrospective review of all retinoblastoma patients treated with intra-arterial melphalan at the University of California, San Francisco from March 2010 to August 2012. Twenty eyes (16 patients) underwent 40 intra-arterial melphalan infusions, and dose was determined by age. Patients were treated at monthly intervals and received a range of 1-5 treatments. Response to therapy, toxicity, and procedural radiation exposure was assessed.
All patients are alive without metastatic disease at a median follow-up of 14.5 (1-29) months. Treatment with enucleation or external beam radiation was avoided in 11/20 eyes (55%) overall [6/12 (50%) in newly diagnosed eyes and 5/8 (63%) in refractory/relapsed eyes]. Response rates (per the International Classification of Retinoblastoma) were as follows: 6/7 (86%) in groups A-C and 5/13 (38%) in groups D and E. Nonhematologic and hematologic toxicities were minimal and comparable with those in previous reports. The mean procedural radiation dose was 20.2 ± 11.9 mGy per eye per procedure.
Superselective intra-arterial melphalan therapy is effective for less advanced eyes but further modifications to therapy are required to improve results in eyes with advanced retinoblastoma.
动脉内注射美法仑化疗在视网膜母细胞瘤的治疗中显示出前景。本报告描述了我们对新诊断的视网膜母细胞瘤患者以及全身化疗后疾病进展接受治疗的患者使用超选择性动脉内注射美法仑的结果。
这是一项对2010年3月至2012年8月在加利福尼亚大学旧金山分校接受动脉内注射美法仑治疗的所有视网膜母细胞瘤患者的回顾性研究。20只眼（16例患者）接受了40次动脉内美法仑输注，剂量根据年龄确定。患者每月接受一次治疗，共接受1 - 5次治疗。评估了治疗反应、毒性和程序性辐射暴露情况。
所有患者在中位随访14.5（1 - 29）个月时均存活且无转移性疾病。总体上，11/20只眼（55%）避免了眼球摘除或外照射治疗[新诊断眼为6/12（50%），难治性/复发性眼为5/8（63%）]。根据视网膜母细胞瘤国际分类，反应率如下：A - C组为6/7（86%），D组和E组为5/13（38%）。非血液学和血液学毒性极小，与既往报告相当。每次手术每只眼的平均程序性辐射剂量为20.2±11.9 mGy。
超选择性动脉内美法仑治疗对病情较轻的眼有效，但需要对治疗进行进一步改进以提高晚期视网膜母细胞瘤眼的治疗效果。
