Details

Autor(en) / 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
• Staszewski, Ori
• Mawrin, Christian
• Schittenhelm, Jens
• Brück, Wolfgang
• Filipski, Katharina
• Hartmann, Christian
• Meinhardt, Matthias
• Pietschmann, Klaus
• Haberler, Christine
• Slavc, Irene
• Gerber, Nicolas U
• Grotzer, Michael
• Benesch, Martin
• Schlegel, Paul-Gerhardt
• Deinlein, Frank
• von Bueren, André O
• Friedrich, Carsten
• Obrecht, Denise
• Fleischhack, Gudrun
• Kwiecien, Robert
• Faldum, Andreas
• Kortmann, Rolf-Dieter
• Kool, Marcel
• Rutkowski, Stefan

Titel

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

Ist Teil von

• Neuro-oncology (Charlottesville, Va.), 2020-12, Vol.22 (Supplement_3), p.iii388-iii389

Ort / Verlag

US: Oxford University Press

Erscheinungsjahr

2020

Link zum Volltext

Quelle

Oxford Journals 2020 Medicine

Beschreibungen/Notizen

• 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.