Packer R J, Siegel K R, Sutton L N, Evans A E, D'Angio G, Rorke L B, Bunin G R, Schut L
Neuro-Oncology Program, Children's Hospital of Philadelphia.
Ann Neurol. 1988 Oct;24(4):503-8. doi: 10.1002/ana.410240405.
Recent studies have demonstrated that preoperative and postoperative factors can broadly stratify patients with medulloblastoma/primitive neuroectodermal tumors (MB/PNET) into risk groups. For children with factors that suggest poor outcome after treatment with surgery and radiotherapy, the addition of chemotherapy can improve survival. Since 1983, 26 children with poor-risk posterior fossa MB/PNET have been treated at our institution with craniospinal radiation therapy and adjuvant chemotherapy. Chemotherapy consisted of vincristine during radiotherapy and eight 6-week cycles of vincristine, cis-platinum, and 1-(2-chloroethyl)-3-cyclohexyl-1-nitrosourea (CCNU). Twenty-five of 26 patients (96%) who have been entered on this protocol remain alive and free of disease at a median of 24 months from diagnosis (range 6 to 50 months). Twenty patients have completed all therapy and are at a median of 32 months from initial diagnosis with no evidence of disease. These patients were compared to a group of children with similar prognostic features treated at our institution between 1975 and 1983. Actuarial disease-free survival was statistically significantly better for protocol patients than for historical control subjects (p less than 0.002). This difference was most marked in patients who had received radiation therapy alone (p less than 0.0003). Actuarial 2-year disease-free survival was 96% for patients on protocol as compared to 59% for historical control patients who had been treated with radiotherapy alone. The chemotherapy given in this protocol was well tolerated. The results of this study, although preliminary, suggest that adjuvant chemotherapy is at least transiently effective in improving the rate of disease-free survival for children with poor-risk MB/PNET.
最近的研究表明，术前和术后因素可大致将髓母细胞瘤/原始神经外胚层肿瘤（MB/PNET）患者分为不同风险组。对于那些经手术和放疗后提示预后不良的儿童，加用化疗可提高生存率。自1983年以来，我院对26例高危后颅窝MB/PNET患儿采用了全脑全脊髓放疗及辅助化疗。化疗方案为放疗期间使用长春新碱，以及8个周期、为期6周的长春新碱、顺铂和1-（2-氯乙基）-3-环己基-1-亚硝基脲（CCNU）联合化疗。纳入该方案的26例患者中有25例（96%）在诊断后中位24个月（范围6至50个月）时仍存活且无疾病。20例患者已完成所有治疗，自初始诊断起中位32个月，无疾病证据。将这些患者与1975年至1983年间在我院接受治疗的一组具有相似预后特征的儿童进行比较。方案组患者的无病生存率经精算在统计学上显著优于历史对照受试者（p<0.002）。这种差异在仅接受放疗的患者中最为明显（p<0.0003）。方案组患者的精算2年无病生存率为96%，而仅接受放疗的历史对照患者为59%。该方案中的化疗耐受性良好。本研究结果虽为初步结果，但表明辅助化疗至少在短期内对提高高危MB/PNET患儿的无病生存率有效。
