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目的探讨大剂量化疗并自体外周血造血干细胞移植(APBSCT)治疗小儿眼外期视网膜母细胞瘤的可行性及疗效。方法对3例视网膜母细胞瘤眼外转移患儿,在常规化疗后进行APBSCT治疗。大剂量化疗(预处理)方案采用卡铂(250 mg/m2,-7~-3 d),依托泊苷(350 mg/m2,-7~-3 d),环磷酰胺(1.6 g/m2,-6~-3 d)。从第0天开始输入自体外周血造血干细胞。结果3例患儿均获得移植成功,外周血白细胞总数>1.0×109/L的平均时间为10 d,中性粒细胞>0.5×109/L的平均时间为12 d,白细胞总数恢复正常的平均时间为15 d,血小板计数>50×109/L的平均时间为23 d;血红蛋白含量平均在移植后第15天升至100 g/L;随访时间6个月~14个月,至今3例患儿均生存。结论大剂量化疗加自体干细胞移植是治疗眼外转移的视网膜母细胞瘤的有效治疗方法。因观察例数和随访时间有限,其远期效果还有待进一步评估。
Objective To investigate the feasibility and efficacy of high-dose chemotherapy combined with autologous peripheral blood stem cell transplantation (APBSCT) in the treatment of extraocular retinoblastoma in children. Methods Three patients with extraocular metastasis of retinoblastoma were treated with APBSCT after routine chemotherapy. High-dose chemotherapy (pretreatment) regimen consisted of carboplatin (250 mg / m2, -7-3 d), etoposide (350 mg / m2, -7-3 d), cyclophosphamide , -6 ~ -3 d). Autologous peripheral blood hematopoietic stem cells are imported from day 0. Results All the 3 children were successfully transplanted. The mean time of leukocyte count> 1.0 × 109 / L was 10 days. The average time of neutrophil> 0.5 × 109 / L was 12 days. The average number of white blood cells returned to normal The average time for platelet count> 50 × 109 / L was 23 days. The average hemoglobin content increased to 100 g / L on the 15th day after transplantation. The follow-up time ranged from 6 months to 14 months. So far, 3 patients Children are living. Conclusion High-dose chemotherapy plus autologous stem cell transplantation is an effective treatment for extraocular metastasis of retinoblastoma. Due to the limited number of cases and follow-up, the long-term effect remains to be evaluated.