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von Bueren, AO; von Hoff, K; Pietsch, T; Gerber, NU; Warmuth-Metz, M; Deinlein, F; Zwiener, I; Faldum, A; Fleischhack, G; Benesch, M; Krauss, J; Kuehl, J; Kortmann, RD; Rutkowski, S.
Treatment of young children with localized medulloblastoma by chemotherapy alone: results of the prospective, multicenter trial HIT 2000 confirming the prognostic impact of histology.
Neuro Oncol. 2011; 13(6):669-679
Doi: 10.1093/neuonc/nor025
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- Co-Autor*innen der Med Uni Graz
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Benesch Martin
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- Abstract:
- This study was designed to confirm the previously observed favorable survival rates and prognostic factors in young children with nonmetastatic medulloblastoma (MB) treated with postoperative chemotherapy alone. Patients who received a diagnosis during the period January 2001 through December 2005 and who were aged <4 years received 3 cycles of postoperative systemic multiagent chemotherapy and intraventricular methotrexate. In cases of complete remission, treatment was terminated after 2 additional cycles of chemotherapy. Otherwise, secondary surgery, radiotherapy, and consolidation chemotherapy were recommended. At a median follow-up of 4.5 years, the 5-year event-free survival (EFS) and overall survival (OS) rates (± standard error) for 45 patients (median age, 2.5 years) were 57% ± 8% and 80% ± 6%, respectively. Nineteen patients with desmoplastic/nodular MB variants had better 5-year EFS and OS rates (90% ± 7% and 100% ± 0%, respectively) than did 23 patients with classic MB (30% ± 11% and 68% ± 10%, respectively; P < .001 for EFS; P = .008 for OS). Five-year EFS and OS rates for 3 children with anaplastic MB were 33% ± 27%. Desmoplastic/nodular histology was an independent prognostic factor for EFS. Twenty-nine of 30 patients without postoperative residual tumor remained in continuous complete remission. Our results confirm that histology of MB variants is a strong prognostic factor in this age group. Sustained tumor control can be achieved by this chemotherapy regimen in young children with desmoplastic/nodular MB variants. For children with non-desmoplastic/nonnodular MB variants, for which predominantly local relapses lead to less favorable survival rates, local radiotherapy has been introduced after chemotherapy since 2006.
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Cerebellar Neoplasms - pathology
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Combined Modality Therapy -
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Follow-Up Studies -
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Humans -
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Methotrexate - administration & dosage
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Prospective Studies -
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brain tumor
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chemotherapy
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children
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medulloblastoma
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treatment