The molecular landscape and associated clinical experience in infant medulloblastoma: prognostic significance of second-generation subtypes.
Infant medulloblastoma
biomarkers
molecular pathology
paediatric oncology
risk stratification
Journal
Neuropathology and applied neurobiology
ISSN: 1365-2990
Titre abrégé: Neuropathol Appl Neurobiol
Pays: England
ID NLM: 7609829
Informations de publication
Date de publication:
02 2021
02 2021
Historique:
received:
05
05
2020
revised:
22
07
2020
accepted:
29
07
2020
pubmed:
12
8
2020
medline:
23
11
2021
entrez:
12
8
2020
Statut:
ppublish
Résumé
Biomarker-driven therapies have not been developed for infant medulloblastoma (iMB). We sought to robustly sub-classify iMB, and proffer strategies for personalized, risk-adapted therapies. We characterized the iMB molecular landscape, including second-generation subtyping, and the associated retrospective clinical experience, using large independent discovery/validation cohorts (n = 387). iMB Investigations of large, retrospective cohorts have enabled the comprehensive and robust characterization of molecular heterogeneity within iMB. Novel subtypes are clinically significant and subgroup-dependent survival models highlight opportunities for biomarker-directed therapies.
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
236-250Subventions
Organisme : Cancer Research UK
ID : C8464/A23391
Pays : United Kingdom
Organisme : Cancer Research UK
ID : 13457
Pays : United Kingdom
Informations de copyright
© 2020 The Authors. Neuropathology and Applied Neurobiology published by John Wiley & Sons Ltd on behalf of British Neuropathological Society.
Références
Pizer BL, Clifford SC. The potential impact of tumour biology on improved clinical practice for medulloblastoma: progress towards biologically driven clinical trials. Br J Neurosurg 2009; 23: 364-75
Jenkin D, Danjoux C, Greenberg M. Subsequent quality of life for children irradiated for a brain tumor before age four years. Med Pediatr Oncol 1998; 31: 506-11
Mulhern RK, Horowitz ME, Kovnar EH, Langston J, Sanford RA, Kun LE. Neurodevelopmental status of infants and young children treated for brain tumors with preirradiation chemotherapy. J Clin Oncol 1989; 7: 1660-6
Louis DN, Perry A, Reifenberger G, von Deimling A, Figarella-Branger D, Cavenee WK, et al. The 2016 World Health Organization Classification of Tumors of the Central Nervous System: a summary. Acta Neuropathol 2016; 131: 803-20
Rutkowski S, von Hoff K, Emser A, Zwiener I, Pietsch T, Figarella-Branger D, et al. Survival and prognostic factors of early childhood medulloblastoma: an international meta-analysis. J Clin Oncol 2010; 28: 4961-8
Northcott PA, Buchhalter I, Morrissy AS, Hovestadt V, Weischenfeldt J, Ehrenberger T, et al. The whole-genome landscape of medulloblastoma subtypes. Nature 2017; 547: 311-7
Robinson GW, Rudneva VA, Buchhalter I, Billups CA, Waszak SM, Smith KS, et al. Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Lancet Oncol 2018; 19: 768-84
Cavalli FMG, Remke M, Rampasek L, Peacock J, Shih DJH, Luu B, et al. Intertumoral Heterogeneity within Medulloblastoma Subgroups. Cancer Cell 2017; 31: 737-54.e6
Schwalbe EC, Lindsey JC, Nakjang S, Crosier S, Smith AJ, Hicks D, et al. Novel molecular subgroups for clinical classification and outcome prediction in childhood medulloblastoma: a cohort study. Lancet Oncol 2017; 18: 958-71
Sharma T, Schwalbe EC, Williamson D, Sill M, Hovestadt V, Mynarek M, et al. Second-generation molecular subgrouping of medulloblastoma: an international meta-analysis of Group 3 and Group 4 subtypes. Acta Neuropathol 2019; 138: 309-26
Clifford SC, Lannering B, Schwalbe EC, Hicks D, O'Toole K, Nicholson SL, et al. Biomarker-driven stratification of disease-risk in non-metastatic medulloblastoma: Results from the multi-center HIT-SIOP-PNET4 clinical trial. Oncotarget 2015; 6: 38827-39
Ellison DW, Kocak M, Dalton J, Megahed H, Lusher ME, Ryan SL, et al. Definition of Disease-Risk Stratification Groups in Childhood Medulloblastoma Using Combined Clinical, Pathologic, and Molecular Variables. J Clin Oncol 2011; 29: 1400-7
Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, et al. Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. J Clin Oncol 2015; 33: 2646-54
Lafay-Cousin L, Bouffet E, Strother D, Rudneva V, Hawkins C, Eberhart C, et al. Phase II Study of Nonmetastatic Desmoplastic Medulloblastoma in Children Younger Than 4 Years of Age: A Report of the Children's Oncology Group (ACNS1221). J Clin Oncol 2020; 38: 223-31
Mynarek M, von Hoff K, Pietsch T, Ottensmeier H, Warmuth-Metz M, Bison B, et al. Nonmetastatic Medulloblastoma of Early Childhood: Results From the Prospective Clinical Trial HIT-2000 and An Extended Validation Cohort. J Clin Oncol 2020; 38: 2028-40
Dhall G, O'Neil SH, Ji L, Haley K, Whitaker AM, Nelson MD, et al. Excellent Outcome of Young Children with Nodular Desmoplastic Medulloblastoma Treated on "Head Start" III: A Multi-Institutional. Prospective Clinical Trial Neuro Oncol 2020. https://doi.org/10.1093/neuonc/noaa102
Schwalbe EC, Williamson D, Lindsey JC, Hamilton D, Ryan SL, Megahed H, et al. DNA methylation profiling of medulloblastoma allows robust subclassification and improved outcome prediction using formalin-fixed biopsies. Acta Neuropathol 2013; 125: 359-71
Hovestadt V, Remke M, Kool M, Pietsch T, Northcott PA, Fischer R, et al. Robust molecular subgrouping and copy-number profiling of medulloblastoma from small amounts of archival tumour material using high-density DNA methylation arrays. Acta Neuropathol 2013; 125: 913-6
Lamont JM, McManamy CS, Pearson AD, Clifford SC, Ellison DW. Combined histopathological and molecular cytogenetic stratification of medulloblastoma patients. Clin Cancer Res. 2004; 10: 5482-93
Langdon JA, Lamont JM, Scott DK, Dyer S, Prebble E, Bown N, et al. Combined genome-wide allelotyping and copy number analysis identify frequent genetic losses without copy number reduction in medulloblastoma. Genes Chromosomes Cancer 2006; 45: 47-60
Goschzik T, Schwalbe EC, Hicks D, Smith A, Zur Muehlen A, Figarella-Branger D, et al. Prognostic effect of whole chromosomal aberration signatures in standard-risk, non-WNT/non-SHH medulloblastoma: a retrospective, molecular analysis of the HIT-SIOP PNET 4 trial. Lancet Oncol 2018; 19: 1602-16
Barone G, Anderson J, Pearson AD, Petrie K, Chesler L. New strategies in neuroblastoma: Therapeutic targeting of MYCN and ALK. Clin Cancer Res 2013; 19: 5814-21