271966 20240918164039.0 10.1093/neuonc/noae071 doi pmid:38578306 pmid pmc:PMC11376455 pmc 1522-8517 ISSN 1523-5866 ISSN DZNE-2024-01108 English 610 Obrecht-Sturm, Denise 0000-0002-3216-8452 0 Treatment response as surrogate to predict risk for disease progression in pediatric medulloblastoma with persistent magnetic resonance imaging lesions after first-line treatment. Oxford 2024 Oxford Univ. Press article DRIVER Output Types/Journal article DataCite Journal Article journal journal PUB:(DE-HGF)16 1726649767_21997 PUB:(DE-HGF) ARTICLE BibTeX JOURNAL_ARTICLE ORCID Journal Article 0 EndNote This study aims at clarifying the impact of persistent residual lesions following first-line treatment for pediatric medulloblastoma.Data on 84 pediatric patients with medulloblastoma and persistent residual lesions on centrally reviewed magnetic resonance imaging (MRI) at the end of first-line therapy were analyzed.Twenty patients (23.8%) had residual lesions in the tumor bed (R+/M0), 51 (60.7%) had distant lesions (R0/M+) and 13 (15.5%) had both (R+/M+). Overall response to first-line therapy was minor or partial (≥ 25% reduction, minor response [MR]/PR) for 64 (76.2%) and stable disease (SD) for 20 patients (23.8%). Five-year post-primary-treatment progression-free (pptPFS) and overall survival (pptOS) were superior after MR/PR (pptPFS: 62.5 ± 7.0%[MR/PR] vs. 35.9 ± 12.8%[SD], P = .03; pptOS: 79.7 ± 5.9[MR/PR] vs. 55.5 ± 13.9[SD], P = .04). Furthermore, R+/M + was associated with a higher risk for progression (5-year pptPFS: 22.9 ± 17.9%[R+, M+] vs. 72.4 ± 12.0%[R+, M0]; P = .03). Watch-and-wait was pursued in 58 patients, while n = 26 received additional treatments (chemotherapy only, n = 19; surgery only, n = 2; combined, n = 3; valproic acid, n = 2), and their outcomes were not superior to watch-and-wait (5-year pptPFS: 58.5 ± 7.7% vs. 51.6 ± 10.7% P = .71; 5-year pptOS: 76.3 ± 6.9% vs. 69.8 ± 9.7%, P = .74). For the whole cohort, 5-year pptPFS by molecular subgroup (58 cases) were WNT: 100%, SHH: 50.0 ± 35.4%, group-4, 52.5 ± 10.5, group-3 54.2 ± 13.8%; (P = .08).Overall response and extent of lesions can function as surrogate parameters to predict outcomes in pediatric MB patients with persistent lesions after first-line therapy. Especially in the case of solitary persistent medulloblastoma MRI lesions, additional therapy was not beneficial. Therefore, treatment response, extent/kind of residual lesions and further diagnostic information need consideration for indication of additional treatments for persisting lesions. 353 - Clinical and Health Care Research (POF4-353) G:(DE-HGF)POF4-353 POF4-353 POF IV 0 Dataset connected to CrossRef, PubMed, , Journals: pub.dzne.de MRI Other children Other medulloblastoma Other persistent residual disease Other Humans MeSH Medulloblastoma: diagnostic imaging MeSH Medulloblastoma: pathology MeSH Male MeSH Child MeSH Female MeSH Magnetic Resonance Imaging: methods MeSH Cerebellar Neoplasms: diagnostic imaging MeSH Cerebellar Neoplasms: pathology MeSH Disease Progression MeSH Child, Preschool MeSH Adolescent MeSH Follow-Up Studies MeSH Prognosis MeSH Retrospective Studies MeSH Survival Rate MeSH Infant MeSH Neoplasm, Residual: diagnostic imaging MeSH Neoplasm, Residual: pathology MeSH Schömig, Lena 1 Mynarek, Martin 0000-0003-3302-2719 2 Bison, Brigitte 3 Schwarz, Rudolf 4 Pietsch, Torsten P:(DE-2719)2812617 5 dzne Pfister, Stefan M 0000-0002-5447-5322 6 Sill, Martin 7 Sturm, Dominik 8 Sahm, Felix 0000-0001-5441-1962 9 Kortmann, Rolf-Dieter 10 Gerber, Nicolas U 11 von Bueren, André O 12 Fleischhack, Gudrun 13 Schüller, Ulrich P:(DE-2719)9000979 14 Nussbaumer, Gunther 15 Benesch, Martin 16 Rutkowski, Stefan 17 10.1093/neuonc/noae071 Vol. 26, no. 9, p. 1712 - 1722 PERI:(DE-600)2094060-9 9 1712 - 1722 Neuro-Oncology 26 2024 1522-8517 http://pub.dzne.de/record/271966/files/DZNE-2024-01108%20SUP.zip http://pub.dzne.de/record/271966/files/DZNE-2024-01108_Restricted.pdf http://pub.dzne.de/record/271966/files/DZNE-2024-01108_Restricted.pdf?subformat=pdfa pdfa oai:pub.dzne.de:271966 VDB Deutsches Zentrum für Neurodegenerative Erkrankungen I:(DE-588)1065079516 DZNE 5 P:(DE-2719)2812617 DE-HGF Gesundheit Neurodegenerative Diseases G:(DE-HGF)POF4-350 G:(DE-HGF)POF4-353 G:(DE-HGF)POF4 G:(DE-HGF)POF4-300 G:(DE-HGF)POF Clinical and Health Care Research 0 2024 Nationallizenz StatID:(DE-HGF)0420 StatID 2023-10-24 ger JCR StatID:(DE-HGF)0100 StatID NEURO-ONCOLOGY : 2022 2023-10-24 DBCoverage StatID:(DE-HGF)0200 StatID SCOPUS 2023-10-24 DBCoverage StatID:(DE-HGF)0300 StatID Medline 2023-10-24 DBCoverage StatID:(DE-HGF)0320 StatID PubMed Central 2023-10-24 DBCoverage StatID:(DE-HGF)0199 StatID Clarivate Analytics Master Journal List 2023-10-24 WoS StatID:(DE-HGF)0113 StatID Science Citation Index Expanded 2023-10-24 DBCoverage StatID:(DE-HGF)0150 StatID Web of Science Core Collection 2023-10-24 DBCoverage StatID:(DE-HGF)0160 StatID Essential Science Indicators 2023-10-24 DBCoverage StatID:(DE-HGF)1110 StatID Current Contents - Clinical Medicine 2023-10-24 IF >= 15 StatID:(DE-HGF)9915 StatID NEURO-ONCOLOGY : 2022 2023-10-24 I:(DE-2719)1011009 Brainbank (Bonn) Brainbank Unit Bonn 0 journal VDB I:(DE-2719)1011009 UNRESTRICTED