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Mynarek, Martin; von Hoff, Katja; Pietsch, Torsten; Ottensmeier, Holger; Warmuth-Metz, Monika; Bison, Brigitte; Pfister, Stefan; Korshunov, Andrey; Sharma, Tanvi; Jaeger, Natalie; Ryzhova, Marina; Zheludkova, Olga; Golanov, Andrey; Rushing, Elisabeth Jane; Hasselblatt, Martin; Koch, Arend; Schüller, Ulrich; von Deimling, Andreas; Sahm, Felix; Sill, Martin; Riemenschneider, Markus J; Dohmen, Hildegard; Monoranu, Camelia-Maria; Sommer, Clemens; [...]

MBCL-07. NON-METASTATIC MEDULLOBLASTOMA OF EARLY CHILDHOOD: RESULTS FROM THE PROSPECTIVE CLINICAL TRIAL HIT-2000 AND AN EXTENDED VALIDATION COHORT

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  • Medientyp: E-Artikel
  • Titel: MBCL-07. NON-METASTATIC MEDULLOBLASTOMA OF EARLY CHILDHOOD: RESULTS FROM THE PROSPECTIVE CLINICAL TRIAL HIT-2000 AND AN EXTENDED VALIDATION COHORT
  • Beteiligte: Mynarek, Martin; von Hoff, Katja; Pietsch, Torsten; Ottensmeier, Holger; Warmuth-Metz, Monika; Bison, Brigitte; Pfister, Stefan; Korshunov, Andrey; Sharma, Tanvi; Jaeger, Natalie; Ryzhova, Marina; Zheludkova, Olga; Golanov, Andrey; Rushing, Elisabeth Jane; Hasselblatt, Martin; Koch, Arend; Schüller, Ulrich; von Deimling, Andreas; Sahm, Felix; Sill, Martin; Riemenschneider, Markus J; Dohmen, Hildegard; Monoranu, Camelia-Maria; Sommer, Clemens; [...]
  • Erschienen: Oxford University Press (OUP), 2020
  • Erschienen in: Neuro-Oncology, 22 (2020) Supplement_3, Seite iii388-iii389
  • Sprache: Englisch
  • DOI: 10.1093/neuonc/noaa222.483
  • ISSN: 1522-8517; 1523-5866
  • Schlagwörter: Cancer Research ; Neurology (clinical) ; Oncology
  • Entstehung:
  • Anmerkungen:
  • Beschreibung: Abstract OBJECTIVE To avoid craniospinal irradiation (CSI) in children younger than four years with non-metastatic medulloblastoma by chemotherapy, intraventricular methotrexate and risk-adapted local radiotherapy. PATIENTS AND METHODS Eighty-seven patients received systemic chemotherapy and intraventricular methotrexate. Until 2006, CSI was reserved for non-response or progression. After 2006, local radiotherapy was introduced for non-responders or classic (CMB), anaplastic or large-cell medulloblastoma (LCA). Infantile SHH-activated medulloblastomas (SHH_INF) were subdivided by DNA-methylation profiling. Survival in SHH_INF subtypes were also assessed in a validation cohort (n=71). RESULTS Patients with desmoplastic medulloblastoma (DMB) or medulloblastoma with extensive nodularity (MBEN) (n=42) had 93% 5-year PFS, 100% 5-year OS and 93% 5-year CSI-free survival. Patients with CMB/LCA (n=45) had 37% 5y-PFS, 62% 5y-OS and 39% 5y-CSI-free survival. Local radiotherapy did not improve survival in CMB/LCA patients. All DMB/MBEN assessed by DNA methylation profiling belonged to the SHH_INF subgroup. Group 3 patients (5y-PFS 36% [n=14]) relapsed more frequently than SHH_INF (5y-PFS 93% [n=28]) or Group 4 patients (5y-PFS 83% [n=6], p<0.001). SHH_INF split into iSHH-I and iSHH-II subtypes in HIT-2000-BIS4 and the validation cohort, without prognostic impact (5y-PFS: iSHH-I 73% vs. iSHH-II 83%, p=0.25, n=99). Mean IQ was 90 (radiotherapy-free survivors) vs. 74 (patients that received CSI) [p=0.012]. CONCLUSION Systemic chemotherapy and intraventricular methotrexate led to favorable survival in both iSHH-subtypes of SHH-activated DMB/MBEN with acceptable neurotoxicity. Survival in non-WNT/non-SHH CMB/LCA patients was not improved by local radiotherapy. Survival was more favorable in patients with Group 4 than in patients with Group 3 medulloblastoma.
  • Zugangsstatus: Freier Zugang