Rutkowski Stefan, Bode Udo, Deinlein Frank, Ottensmeier Holger, Warmuth-Metz Monika, Soerensen Niels, Graf Norbert, Emser Angela, Pietsch Torsten, Wolff Johannes E A, Kortmann Rolf D, Kuehl Joachim
Department of Pediatric Oncology, Children's Hospital, University of Würzburg, Würzburg, Germany.
N Engl J Med. 2005 Mar 10;352(10):978-86. doi: 10.1056/NEJMoa042176.
The prognosis for young children with medulloblastoma is poor, and survivors are at high risk for cognitive deficits. We conducted a trial of the treatment of this brain tumor by intensive postoperative chemotherapy alone.
After surgery, children received three cycles of intravenous chemotherapy (cyclophosphamide, vincristine, methotrexate, carboplatin, and etoposide) and intraventricular methotrexate. Treatment was terminated if a complete remission was achieved. Leukoencephalopathy and cognitive deficits were evaluated.
Forty-three children were treated according to protocol. In children who had complete resection (17 patients), residual tumor (14), and macroscopic metastases (12), the five-year progression-free and overall survival rates (+/-SE) were 82+/-9 percent and 93+/-6 percent, 50+/-13 percent and 56+/-14 percent, and 33+/-14 percent and 38+/-15 percent, respectively. The rates in 31 patients without macroscopic metastases were 68+/-8 percent and 77+/-8 percent. Desmoplastic histology, metastatic disease, and an age younger than two years were independent prognostic factors for tumor relapse and survival. Treatment strategies at relapse were successful in 8 of 16 patients. There were no major instances of unexpected toxicity. In 19 of 23 children, asymptomatic leukoencephalopathy was detected by magnetic resonance imaging. After treatment, the mean IQ was significantly lower than that of healthy controls within the same age group but higher than that of patients in a previous trial who had received radiotherapy.
Postoperative chemotherapy alone is a promising treatment for medulloblastoma in young children without metastases.
小儿髓母细胞瘤预后较差，幸存者存在认知缺陷的高风险。我们开展了一项仅采用强化术后化疗治疗这种脑肿瘤的试验。
术后，儿童接受三个周期的静脉化疗（环磷酰胺、长春新碱、甲氨蝶呤、卡铂和依托泊苷）及脑室内甲氨蝶呤治疗。若实现完全缓解则终止治疗。对脑白质病和认知缺陷进行评估。
43名儿童按方案接受治疗。在肿瘤完全切除的儿童（17例）、有残留肿瘤的儿童（14例）和有肉眼可见转移灶的儿童（12例）中，五年无进展生存率和总生存率（±标准误）分别为82±9%和93±6%、50±13%和56±14%、33±14%和38±15%。31例无肉眼可见转移灶的患者的生存率分别为68±8%和77±8%。促纤维增生性组织学、转移疾病和年龄小于2岁是肿瘤复发和生存的独立预后因素。16例复发患者中有8例的复发治疗策略成功。未出现严重的意外毒性反应。23名儿童中有19名通过磁共振成像检测到无症状性脑白质病。治疗后，平均智商显著低于同年龄组的健康对照，但高于先前接受放疗的试验患者。
对于无转移的小儿髓母细胞瘤，单纯术后化疗是一种有前景的治疗方法。
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