Dunkel I J, Boyett J M, Yates A, Rosenblum M, Garvin J H, Bostrom B C, Goldman S, Sender L S, Gardner S L, Li H, Allen J C, Finlay J L
Memorial Sloan-Kettering Cancer Center, New York, NY, USA.
J Clin Oncol. 1998 Jan;16(1):222-8. doi: 10.1200/JCO.1998.16.1.222.
Medulloblastoma is a highly lethal disease when it recurs. Very few patients survive with conventional treatment. This study evaluated the use of high-dose carboplatin, thiotepa, and etoposide with autologous stem-cell rescue (ASCR) in patients with recurrent medulloblastoma.
Chemotherapy consisted of carboplatin 500 mg/m2 (or area under the curve = 7 mg/mL x min via Calvert formula) on days -8, -7, and -6; and thiotepa 300 mg/m2 and etoposide 250 mg/m2 on days -5, -4, and -3; followed by ASCR on day 0. In addition to the study-prescribed therapy, 21 patients received other treatment: neurosurgical resection in seven, conventional chemotherapy in 17, and external-beam irradiation in 11 cases.
Twenty-three patients with recurrent medulloblastoma, aged two to 44 years (median, 13 years) at ASCR, were treated. Three patients died of treatment-related toxicities within 21 days of ASCR; multiorgan system failure in two, and Aspergillus infection with venoocclusive disease in one. Seven of 23 patients (30%) are event-free survivors at a median of 54 months post-ASCR (range, 24 to 78 months). Kaplan-Meier estimates of event-free (EFS) and overall survival are 34% +/- 10% and 46% +/- 11%, respectively, at 36 months post-ASCR.
This strategy may provide long-term survival for some patients with recurrent medulloblastoma.
髓母细胞瘤复发时是一种高致死性疾病。采用传统治疗方法时极少有患者存活。本研究评估了大剂量卡铂、噻替派和依托泊苷联合自体干细胞救援（ASCR）在复发性髓母细胞瘤患者中的应用。
化疗方案为在第-8、-7和-6天给予卡铂500mg/m²（或根据卡尔弗特公式计算曲线下面积=7mg/mL×min）；在第-5、-4和-3天给予噻替派300mg/m²和依托泊苷250mg/m²；然后在第0天进行ASCR。除了研究规定的治疗外，21例患者接受了其他治疗：7例接受了神经外科手术切除，17例接受了传统化疗，11例接受了外照射。
对23例复发性髓母细胞瘤患者进行了治疗，ASCR时年龄为2至44岁（中位数为13岁）。3例患者在ASCR后21天内死于治疗相关毒性；2例死于多器官系统衰竭，1例死于曲霉菌感染合并肝静脉闭塞病。23例患者中有7例（30%）在ASCR后中位54个月（范围为24至78个月）时无事件生存。ASCR后36个月时，无事件生存（EFS）和总生存的Kaplan-Meier估计值分别为34%±10%和46%±11%。
该策略可能为部分复发性髓母细胞瘤患者提供长期生存。
