自体造血干细胞移植治疗转移性视网膜母细胞瘤患儿5例临床效果观察

Clinical analysis of five childhood metastatic retinoblastoma treated with autologous stem cell transplantation

目的分析经自体造血干细胞移植(Auto-HSCT)治疗的转移性视网膜母细胞瘤(RB)患儿的临床资料,探讨其临床特点、治疗方法及预后情况。方法回顾性分析我院收治的5例经Auto-HSCT治疗的转移性RB患儿的临床资料。结果5例RB患儿中男2例,女3例;2例为单眼发病,3例为双眼发病。所有患者均在转移后接受了包括全身静脉大剂量化疗联合自体干细胞移植治疗在内的综合治疗,所有患儿均达到完全缓解,但2例中枢神经系统(CNS)转移患儿分别在移植后的2个月、6个月出现复发,最终均死于颅内转移,另3例非CNS转移患儿无病生存至今。结论转移性RB预后极差,Auto-HSCT和其他综合治疗可能改善非CNS转移者的预后,但仍需警惕CNS转移患儿治疗后的复发。

Objective To analyze the clinical data of children with metastatic retinoblastoma(RB)treated with autologous hematopoietic stem cell transplantation(Auto-HSCT),and to explore its clinical characteristics,treatment and prognosis.Methods Clinical data of five metastatic RB children received auto-HSCT in our hospital were analyzed retrospectively.Results Among 5 RB patients,there were 2 males and 3 females,2 cases had unilateral disease,3 cases had bilateral disease.After metastasis,all patients received comprehensive treatment including systemic intravenous high-dose chemotherapy and auto-HSCT.All patients achieved complete remission,but 2 patients had central nervous system(CNS)metastasis relapsed at 2 months and 6 months after transplantation,respectively,and eventually died of intracranial metastasis,and the other 3 children without CNS metastasis survived and still be disease-free so far.Conclusions The prognosis of metastatic RB is very poor.Auto-HSCT and other comprehensive treatment may improve the prognosis of patients with non-CNS metastases,but the recurrence for patients with CNS metastases after treatment should still be vigilant.