自体外周血干细胞移植治疗儿童视网膜母细胞瘤伴广泛脑室内转移1例并文献复习

目的探讨自体外周血干细胞移植(ABSCT)联合持续侧脑室引流、脑室内给药对儿童视网膜母细胞瘤(RB)广泛脑室内转移的治疗效果。方法 1例RB广泛脑室内转移的7岁患儿,脑脊液病理可见肿瘤细胞满视野,行ABSCT治疗。预处理方案:卡铂+依托泊苷+环磷酰胺,同时予持续侧脑室引流,经引流管侧脑室给药(甲氨蝶呤+地塞米松)。0 d输入自体外周血造血干细胞。结果 +11 d始,脑脊液找瘤细胞持续转阴,+17 d,骨髓造血重建,+24 d,拔除侧脑室引流管,脑脊液神经元特异性烯醇化酶(NSE)及蛋白均降至正常,+36 d复查头颅增强MRI,颅内病灶消失。结论 ABSCT联合持续侧脑室引流脑室内给药治疗RB广泛脑室内转移,对于RB颅内转移患儿的治疗有一定借鉴作用。

Treatment of childhood retinoblastoma widespread encephalocoele metastasis by autologous peripheral blood stem cell transplantation

Objective To investigate the therapeutic effect of autologous peripheral blood stem cell transplantation(ABSCT) combined with continual ventricle drainage and intraventricular administration in patients with retinoblastoma(RB) accompanied by extensive intraventricular metastasis.Methods A 7-year-old child with RB accompanied by widespread encephalocoele metastasis received ABSCT,whose cerebrospinal fluid examination showed full view of tumor cells.The strategy of pre-treatment was as follows: CBP+VP-16+CTX as well as ventricle drainage and lateral cerebral ventricle injection(MTX+dexamethasone).After pre-treatment,autologous peripheral blood stem cells were transplanted into the patient.Results The tumor cells sustained negative in cerebrospinal fluid on the 11rd day.The bone marrow hematopoietic function was reconstructed on the 17th day.The drainage tubes were removed and the examinations of neuron-specific enolase(NSE) and proteins in cerebrospinal fluid returned to normal on the 24th day.The cranial enhanced magnetic resonance recheck showed that the intracranial lesions disappeared.Conclusions Such a rare case report in China showed an effective treatment of ABSCT plus continual ventricle drainage and intraventricular administration in RB combined with extensive brain metastasis,which prolonged the patient’s life,earned more time for the further treatment and provided valuable experience for the treatment of RB with brain metastases.