TY  - JOUR
AU  - Mynarek, Martin
AU  - von Hoff, Katja
AU  - Pietsch, Torsten
AU  - Ottensmeier, Holger
AU  - Warmuth-Metz, Monika
AU  - Bison, Brigitte
AU  - Pfister, Stefan
AU  - Korshunov, Andrey
AU  - Sharma, Tanvi
AU  - Jaeger, Natalie
AU  - Ryzhova, Marina
AU  - Zheludkova, Olga
AU  - Golanov, Andrey
AU  - Rushing, Elisabeth Jane
AU  - Hasselblatt, Martin
AU  - Koch, Arend
AU  - Schüller, Ulrich
AU  - von Deimling, Andreas
AU  - Sahm, Felix
AU  - Sill, Martin
AU  - Riemenschneider, Markus J.
AU  - Dohmen, Hildegard
AU  - Monoranu, Camelia Maria
AU  - Sommer, Clemens
AU  - Staszewski, Ori
AU  - Mawrin, Christian
AU  - Schittenhelm, Jens
AU  - Brück, Wolfgang
AU  - Filipski, Katharina
AU  - Hartmann, Christian
AU  - Meinhardt, Matthias
AU  - Pietschmann, Klaus
AU  - Haberler, Christine
AU  - Slavc, Irene
AU  - Gerber, Nicolas U.
AU  - Grotzer, Michael
AU  - Benesch, Martin
AU  - Schlegel, Paul Gerhardt
AU  - Deinlein, Frank
AU  - von Bueren, André O.
AU  - Friedrich, Carsten
AU  - Juhnke, Björn-Ole
AU  - Obrecht, Denise
AU  - Fleischhack, Gudrun
AU  - Kwiecien, Robert
AU  - Faldum, Andreas
AU  - Kortmann, Rolf Dieter
AU  - Kool, Marcel
AU  - Rutkowski, Stefan
TI  - Nonmetastatic Medulloblastoma of Early Childhood: Results From the Prospective Clinical Trial HIT-2000 and An Extended Validation Cohort
JO  - Journal of clinical oncology
VL  - 38
IS  - 18
SN  - 1527-7755
CY  - Alexandria, Va.
PB  - American Society of Clinical Oncology
M1  - DZNE-2020-01353
SP  - 2028 - 2040
PY  - 2020
AB  - PURPOSEThe HIT-2000-BIS4 trial aimed to avoid highly detrimental craniospinal irradiation (CSI) in children < 4 years of age with nonmetastatic medulloblastoma by systemic chemotherapy, intraventricular methotrexate, and risk-adapted local radiotherapy.PATIENTS AND METHODSFrom 2001-2011, 87 patients received systemic chemotherapy and intraventricular methotrexate. Until 2006, CSI was reserved for nonresponse or progression. After 2006, local radiotherapy was introduced for nonresponders or patients with classic medulloblastoma (CMB) or large-cell/anaplastic medulloblastoma (LCA). DNA methylation profiles of infantile sonic hedgehog-activated medulloblastoma (SHH-INF) were subdivided into iSHH-I and iSHH-II subtypes in the HIT-2000-BIS4 cohort and a validation cohort (n = 71) from the HIT group and Russia.RESULTSFive years after diagnosis, patients with desmoplastic medulloblastoma (DMB) or medulloblastoma with extensive nodularity (MBEN; n = 42) had 93
KW  - Cerebellar Neoplasms: drug therapy
KW  - Cerebellar Neoplasms: mortality
KW  - Cerebellar Neoplasms: radiotherapy
KW  - Child, Preschool
KW  - Cranial Irradiation: adverse effects
KW  - DNA Methylation
KW  - Female
KW  - Humans
KW  - Infant
KW  - Male
KW  - Medulloblastoma: drug therapy
KW  - Medulloblastoma: mortality
KW  - Medulloblastoma: radiotherapy
KW  - Methotrexate: administration & dosage
KW  - Neuropsychological Tests
KW  - Prospective Studies
LB  - PUB:(DE-HGF)16
C6  - pmid:32330099
DO  - DOI:10.1200/JCO.19.03057
UR  - https://pub.dzne.de/record/153356
ER  -