自体外周血造血干细胞移植在小儿视网膜母细胞瘤的临床应用

Effect of autologous peripheral blood stem cell transplantation on retinoblastoma in children

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 effect of high dose chemotherapy accompanied with autologous peripheral blood stem cell transplantation（APBSCT） in the treatment of children with extraocular retinoblastoma.Methods Three patients with extraocular retinoblastoma and aged from 17 to 48 months,were given high dose chemotherapy with APBSCT.The pretreatment regimen include carboplatin（250 mg/m2,-7^-3 d）,Etoposide（350 mg/m2,-7^-3 d） and Cyclophosphamide（1.6 g/m2,-6^-3 d）.Peripheral hematopoietic stem cell were thawed and infused on day 0.Results All cases got successful engraftment.It took 10 days on average for peripheral leucocyte count to reach 1.0×109/L and 15 days to 4.0×109/L.12 days were needed for neutrophils to regain 0.5×109/L,23 days for platelets to achieve 50×109/L and 15 days for hemoglobin to 100 g/L.All cases were survival after following up for 6~14 months.Conclusion High dose chemotherapy with APBSCT is effective in patients with metastatic retinoblastoma.Because of the small sample size and the short time of observation,the long term effect should be followed up.