163608 20240320115520.0 10.1007/s11060-021-03913-5 doi pmid:35190934 pmid pmc:PMC8938370 pmc 0167-594X ISSN 1573-7373 ISSN altmetric:123513577 altmetric DZNE-2022-00354 English 610 Obrecht, Denise 0 Clinical and molecular characterization of isolated M1 disease in pediatric medulloblastoma: experience from the German HIT-MED studies. Dordrecht [u.a.] 2022 Springer Science + Business Media B.V article DRIVER Output Types/Journal article DataCite Journal Article journal journal PUB:(DE-HGF)16 1655198547_924 PUB:(DE-HGF) ARTICLE BibTeX JOURNAL_ARTICLE ORCID Journal Article 0 EndNote (CC BY) To evaluate the clinical impact of isolated spread of medulloblastoma cells into cerebrospinal fluid without additional macroscopic metastases (M1-only).The HIT-MED database was searched for pediatric patients with M1-only medulloblastoma diagnosed from 2000 to 2019. Corresponding clinical and molecular data was evaluated. Treatment was stratified by age and changed over time for older patients.70 patients with centrally reviewed M1-only disease were identified. Clinical data was available for all and molecular data for 45/70 cases. 91% were non-WNT/non-SHH medulloblastoma (Grp3/4). 5-year PFS for 52 patients ≥ 4 years was 59.4 (± 7.1) %, receiving either upfront craniospinal irradiation (CSI) or SKK-sandwich chemotherapy (CT). Outcomes did not differ between these strategies (5-year PFS: CSI 61.7 ± 9.9%, SKK-CT 56.7 ± 6.1%). For patients < 4 years (n = 18), 5-year PFS was 50.0 (± 13.2) %. M1-persistence occurred exclusively using postoperative CT and was a strong negative predictive factor (pPFS/OS < 0.01). Patients with additional clinical or molecular high-risk (HR) characteristics had worse outcomes (5-year PFS 42.7 ± 10.6% vs. 64.0 ± 7.0%, p = 0.03). In n = 22 patients ≥ 4 years with full molecular information and without additional HR characteristics, risk classification by molecular subtyping had an effect on 5-year PFS (HR 16.7 ± 15.2%, SR 77.8 ± 13.9%; p = 0.01).Our results confirm that M1-only is a high-risk condition, and further underline the importance of CSF staging. Specific risk stratification of affected patients needs attention in future discussions for trials and treatment recommendations. Future patients without contraindications may benefit from upfront CSI by sparing risks related to higher cumulative CT applied in sandwich regimen. 354 - Disease Prevention and Healthy Aging (POF4-354) G:(DE-HGF)POF4-354 POF4-354 POF IV 0 Dataset connected to CrossRef, PubMed, , Journals: pub.dzne.de Cerebrospinal fluid Other Children Other Medulloblastoma Other Metastases Other Radiotherapy Other Cerebellar Neoplasms: drug therapy MeSH Cerebellar Neoplasms: therapy MeSH Child MeSH Craniospinal Irradiation MeSH Humans MeSH Medulloblastoma: drug therapy MeSH Medulloblastoma: therapy MeSH Risk Factors MeSH Mynarek, Martin 1 Hagel, Christian 2 Kwiecien, Robert 3 Spohn, Michael 4 Bockmayr, Michael 5 Bison, Brigitte 6 Pfister, Stefan M 7 Jones, David T W 8 Sturm, Dominik 9 von Deimling, Andreas 10 Sahm, Felix 11 von Hoff, Katja 12 Juhnke, B-Ole 13 Benesch, Martin 14 Gerber, Nicolas U 15 Friedrich, Carsten 16 von Bueren, André O 17 Kortmann, Rolf-Dieter 18 Schwarz, Rudolf 19 Pietsch, Torsten P:(DE-2719)2812617 20 dzne Fleischhack, Gudrun 21 Schüller, Ulrich P:(DE-2719)9000979 22 dzne Rutkowski, Stefan 0000-0002-5446-9571 23 10.1007/s11060-021-03913-5 Vol. 157, no. 1, p. 37 - 48 PERI:(DE-600)2007293-4 1 37 - 48 Journal of neuro-oncology 157 2022 0167-594X OpenAccess http://pub.dzne.de/record/163608/files/DZNE-2022-00354.pdf OpenAccess pdfa http://pub.dzne.de/record/163608/files/DZNE-2022-00354.pdf?subformat=pdfa oai:pub.dzne.de:163608 openaire open_access VDB driver dnbdelivery Deutsches Zentrum für Neurodegenerative Erkrankungen I:(DE-588)1065079516 DZNE 20 P:(DE-2719)2812617 External Institute I:(DE-HGF)0 Extern 22 P:(DE-2719)9000979 DE-HGF Gesundheit Neurodegenerative Diseases G:(DE-HGF)POF4-350 G:(DE-HGF)POF4-354 G:(DE-HGF)POF4 G:(DE-HGF)POF4-300 G:(DE-HGF)POF Disease Prevention and Healthy Aging 0 2022 DBCoverage StatID:(DE-HGF)0200 StatID SCOPUS 2022-11-08 DBCoverage StatID:(DE-HGF)0300 StatID Medline 2022-11-08 Creative Commons Attribution CC BY 4.0 LIC:(DE-HGF)CCBY4 HGFVOC JCR StatID:(DE-HGF)0100 StatID J NEURO-ONCOL : 2021 2022-11-08 WoS StatID:(DE-HGF)0113 StatID Science Citation Index Expanded 2021-01-28 DEAL Springer StatID:(DE-HGF)3002 StatID 2021-01-28 ger OpenAccess StatID:(DE-HGF)0510 StatID DBCoverage StatID:(DE-HGF)0150 StatID Web of Science Core Collection 2022-11-08 IF < 5 StatID:(DE-HGF)9900 StatID 2022-11-08 DBCoverage StatID:(DE-HGF)0160 StatID Essential Science Indicators 2021-01-28 DBCoverage StatID:(DE-HGF)1110 StatID Current Contents - Clinical Medicine 2022-11-08 Nationallizenz StatID:(DE-HGF)0420 StatID 2022-11-08 ger DBCoverage StatID:(DE-HGF)0199 StatID Clarivate Analytics Master Journal List 2022-11-08 I:(DE-2719)1011009 Brainbank Unit Bonn Brainbank Unit Bonn 0 journal VDB UNRESTRICTED I:(DE-2719)1011009 FullTexts