Treatment of early childhood medulloblastoma by postoperative chemotherapy alone

被引:518
作者
Rutkowski, S
Bode, U
Deinlein, F
Ottensmeier, H
Warmuth-Metz, M
Soerensen, N
Graf, N
Emser, A
Pietsch, T
Wolff, JEA
Kortmann, RD
Kuehl, J
机构
[1] Univ Wurzburg, Dept Neuroradiol, Wurzburg, Germany
[2] Univ Wurzburg, Dept Pediat Neurosurg, Wurzburg, Germany
[3] Univ Bonn, Dept Neuropathol, Natl Reference Ctr Brain Tumors, D-5300 Bonn, Germany
[4] Univ Bonn, Childrens Hosp, Dept Pediat Oncol, D-5300 Bonn, Germany
[5] Univ Homburg, Childrens Hosp, Dept Pediat Oncol, D-6650 Homburg, Germany
[6] Univ Mainz, Inst Med Stat Epidemiol & Informat, D-6500 Mainz, Germany
[7] Univ Regensburg, Childrens Hosp, Dept Pediat Oncol, D-8400 Regensburg, Germany
[8] Univ Leipzig, Dept Radiotherapy, D-7010 Leipzig, Germany
关键词
D O I
10.1056/NEJMoa042176
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: Postoperative chemotherapy alone is a promising treatment for medulloblastoma in young children without metastases.
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页码:978 / 986
页数:9
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