Memorial Sloan-Kettering Cancer Center, New York, New York 10065, USA.
Pediatr Blood Cancer. 2010 Jul 15;55(1):149-52. doi: 10.1002/pbc.22491.
Stage 4b retinoblastoma (central nervous system metastatic disease) has been lethal in virtually all cases reported. Here we describe a series of eight patients treated with intensive chemotherapy, defined as the intention to include high-dose chemotherapy with autologous hematopoietic stem cell rescue.
Induction chemotherapy included cyclophosphamide and/or carboplatin with a topoisomerase inhibitor. High-dose chemotherapy regimens were carboplatin and thiotepa with or without etoposide (n = 3) or carboplatin, etoposide, and cyclophosphamide (n = 2).
Seven patients had leptomeningeal disease and one patient had only direct extension to the CNS via the optic nerve. Three patients had stage 4b disease at the time of original diagnosis of the intra-ocular retinoblastoma; five had later onset at a median of 12 months (range 3-69 months). One patient died of toxicity (septicemia and multi-organ system failure) during induction and two had disease progression prior to high-dose chemotherapy. Five patients received high-dose chemotherapy at a median of 6 months (range 4-6) post-diagnosis of stage 4b disease. Two patients survive event-free at 40 and 101 months; one was irradiated following recovery from the high-dose chemotherapy.
Intensive multimodality therapy may be beneficial for some patients with stage 4b retinoblastoma. Longer follow-up will determine whether it has been curative.
四期 b 型视网膜母细胞瘤（中枢神经系统转移疾病）在几乎所有报告的病例中均致命。在此，我们描述了一组 8 例接受强化化疗治疗的患者，该化疗方案被定义为包括大剂量化疗联合自体造血干细胞挽救治疗的意向。
诱导化疗包括环磷酰胺和/或卡铂联合拓扑异构酶抑制剂。大剂量化疗方案为卡铂和噻替哌联合或不联合依托泊苷（n = 3）或卡铂、依托泊苷和环磷酰胺（n = 2）。
7 例患者有脑膜疾病，1 例患者仅通过视神经直接向 CNS 延伸。3 例患者在初次诊断为眼内视网膜母细胞瘤时已处于四期 b 期；5 例患者在中位时间 12 个月（范围 3-69 个月）后发病。1 例患者在诱导期因毒性（败血症和多器官系统衰竭）死亡，2 例患者在接受大剂量化疗前出现疾病进展。5 例患者在四期 b 期诊断后中位时间 6 个月（范围 4-6）接受大剂量化疗。2 例患者无事件生存，分别为 40 个月和 101 个月；1 例患者在大剂量化疗后接受放疗。
强化多模式治疗可能对某些四期 b 型视网膜母细胞瘤患者有益。更长时间的随访将确定其是否具有治愈性。
