Treatment of young children with localized medulloblastoma by chemotherapy alone: results of the prospective, multicenter trial HIT 2000 confirming the prognostic impact of histology.
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Treatment of young children with localized medulloblastoma by chemotherapy alone: results of the prospective, multicenter trial HIT 2000 confirming the prognostic impact of histology. / von Bueren, André; von Hoff, Katja; von Hoff, Katja; Pietsch, Torsten; Gerber, Nicolas U; Warmuth-Metz, Monika; Deinlein, Frank; Zwiener, Isabella; Faldum, Andreas; Fleischhack, Gudrun; Benesch, Martin; Krauss, Juergen; Kuehl, Joachim; Rutkowski, Stefan; Rutkowski, Stefan.
In: NEURO-ONCOLOGY, Vol. 13, No. 6, 6, 2011, p. 669-679.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Treatment of young children with localized medulloblastoma by chemotherapy alone: results of the prospective, multicenter trial HIT 2000 confirming the prognostic impact of histology.
AU - von Bueren, André
AU - von Hoff, Katja
AU - von Hoff, Katja
AU - Pietsch, Torsten
AU - Gerber, Nicolas U
AU - Warmuth-Metz, Monika
AU - Deinlein, Frank
AU - Zwiener, Isabella
AU - Faldum, Andreas
AU - Fleischhack, Gudrun
AU - Benesch, Martin
AU - Krauss, Juergen
AU - Kuehl, Joachim
AU - Rutkowski, Stefan
AU - Rutkowski, Stefan
PY - 2011
Y1 - 2011
N2 - 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.
AB - 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.
KW - Humans
KW - Male
KW - Female
KW - Prospective Studies
KW - Prognosis
KW - Survival Rate
KW - Follow-Up Studies
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - Magnetic Resonance Imaging
KW - Remission Induction
KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use
KW - Carboplatin/administration & dosage
KW - Cerebellar Neoplasms/pathology/therapy
KW - Cyclophosphamide/administration & dosage
KW - Etoposide/administration & dosage
KW - Leucovorin/administration & dosage
KW - Medulloblastoma/pathology/therapy
KW - Methotrexate/administration & dosage
KW - Neoplasm Recurrence, Local/pathology/therapy
KW - Radiotherapy
KW - Vincristine/administration & dosage
KW - Humans
KW - Male
KW - Female
KW - Prospective Studies
KW - Prognosis
KW - Survival Rate
KW - Follow-Up Studies
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - Magnetic Resonance Imaging
KW - Remission Induction
KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use
KW - Carboplatin/administration & dosage
KW - Cerebellar Neoplasms/pathology/therapy
KW - Cyclophosphamide/administration & dosage
KW - Etoposide/administration & dosage
KW - Leucovorin/administration & dosage
KW - Medulloblastoma/pathology/therapy
KW - Methotrexate/administration & dosage
KW - Neoplasm Recurrence, Local/pathology/therapy
KW - Radiotherapy
KW - Vincristine/administration & dosage
M3 - SCORING: Journal article
VL - 13
SP - 669
EP - 679
JO - NEURO-ONCOLOGY
JF - NEURO-ONCOLOGY
SN - 1522-8517
IS - 6
M1 - 6
ER -