TY - JOUR AU - Gajjar, Amar AU - Robinson, Giles W AU - Smith, Kyle S AU - Lin, Tong AU - Merchant, Thomas E AU - Chintagumpala, Murali AU - Mahajan, Anita AU - Su, Jack AU - Bouffet, Eric AU - Bartels, Ute AU - Schechter, Tal AU - Hassall, Tim AU - Robertson, Thomas AU - Nicholls, Wayne AU - Gururangan, Sridharan AU - Schroeder, Kristin AU - Sullivan, Michael AU - Wheeler, Greg AU - Hansford, Jordan R AU - Kellie, Stewart J AU - McCowage, Geoffrey AU - Cohn, Richard AU - Fisher, Michael J AU - Krasin, Matthew J AU - Stewart, Clinton F AU - Broniscer, Alberto AU - Buchhalter, Ivo AU - Tatevossian, Ruth G AU - Orr, Brent A AU - Neale, Geoff AU - Klimo, Paul AU - Boop, Frederick AU - Srinivasan, Ashok AU - Pfister, Stefan M AU - Gilbertson, Richard J AU - Onar-Thomas, Arzu AU - Ellison, David W AU - Northcott, Paul A TI - Outcomes by Clinical and Molecular Features in Children With Medulloblastoma Treated With Risk-Adapted Therapy: Results of an International Phase III Trial (SJMB03). JO - Journal of clinical oncology VL - 39 IS - 7 SN - 1527-7755 CY - Alexandria, Va. PB - American Society of Clinical Oncology M1 - DKFZ-2021-00042 SP - 822-835 PY - 2021 N1 - 2021 Mar 1;39(7):822-835 AB - SJMB03 (ClinicalTrials.gov identifier: NCT00085202) was a phase III risk-adapted trial that aimed to determine the frequency and clinical significance of biological variants and genetic alterations in medulloblastoma.Patients 3-21 years old were stratified into average-risk and high-risk treatment groups based on metastatic status and extent of resection. Medulloblastomas were molecularly classified into subgroups (Wingless [WNT], Sonic Hedgehog [SHH], group 3, and group 4) and subtypes based on DNA methylation profiles and overlaid with gene mutations from next-generation sequencing. Coprimary study end points were (1) to assess the relationship between ERBB2 protein expression in tumors and progression-free survival (PFS), and (2) to estimate the frequency of mutations associated with WNT and SHH tumors. Clinical and molecular risk factors were evaluated, and the most robust were used to model new risk-classification categories.Three hundred thirty eligible patients with medulloblastoma were enrolled. Five-year PFS was 83.2 LB - PUB:(DE-HGF)16 C6 - pmid:33405951 DO - DOI:10.1200/JCO.20.01372 UR - https://inrepo02.dkfz.de/record/166689 ER -