TY - JOUR AU - Hwang, Eugene I AU - Kool, Marcel AU - Burger, Peter C AU - Capper, David AU - Chavez, Lukas AU - Brabetz, Sebastian AU - Williams-Hughes, Chris AU - Billups, Catherine AU - Heier, Linda AU - Jaju, Alok AU - Michalski, Jeff AU - Li, Yimei AU - Leary, Sarah AU - Zhou, Tianni AU - von Deimling, Andreas AU - Jones, David AU - Fouladi, Maryam AU - Pollack, Ian F AU - Gajjar, Amar AU - Packer, Roger J AU - Pfister, Stefan AU - Olson, James M TI - Extensive Molecular and Clinical Heterogeneity in Patients With Histologically Diagnosed CNS-PNET Treated as a Single Entity: A Report From the Children's Oncology Group Randomized ACNS0332 Trial. JO - Journal of clinical oncology VL - 36 IS - 34 SN - 1527-7755 CY - Alexandria, Va. PB - American Society of Clinical Oncology M1 - DKFZ-2018-01724 SP - 3388 PY - 2018 AB - Children with histologically diagnosed high-risk medulloblastoma, supratentorial primitive neuroectodermal tumor of the CNS (CNS-PNET), and pineoblastoma (PBL) have had poor survival despite intensive treatment. We included these patients in this Children's Oncology Group trial. Molecular profiling later revealed tumor heterogeneity that was not detectable at protocol inception. Enrollment of patients with CNS-PNET/PBL was subsequently discontinued, and outcomes for this part of the study are reported here.In this phase III, four-arm prospective trial, consenting children age 3-22 years with newly diagnosed CNS-PNET were randomly assigned (1:1) to receive carboplatin during radiation and/or adjuvant isotretinoin after standard intensive therapy. Primary outcome measure was event-free survival (EFS) in the intent-to-treat population. Molecular tumor classification was retrospectively completed using DNA methylation profiling.Eighty-five participants with institutionally diagnosed CNS-PNETs/PBLs were enrolled. Of 60 patients with sufficient tissue, 31 were nonpineal in location, of which 22 (71 LB - PUB:(DE-HGF)16 C6 - pmid:30332335 DO - DOI:10.1200/JCO.2017.76.4720 UR - https://inrepo02.dkfz.de/record/141201 ER -