Atypical Teratoid Rhabdoid Tumour : From Tumours to Therapies
- Author:
Elizabeth Anne RICHARDSON
1
;
Ben HO
;
Annie HUANG
Author Information
- Publication Type:Review
- Keywords: Brain neoplasms; Rhabdoid tumour; SMARCB1 protein, Human; Antineoplastic agent; Epigenomics; Protein-tyrosine kinases
- MeSH: Brain; Brain Neoplasms; Central Nervous System; Child; Chromatin Assembly and Disassembly; Drug Therapy; Epigenomics; Humans; Phosphotransferases; Protein-Tyrosine Kinases; Radiotherapy; Stem Cells; Tyrosine
- From:Journal of Korean Neurosurgical Society 2018;61(3):302-311
- CountryRepublic of Korea
- Language:English
- Abstract: Atypical teratoid rhabdoid tumours (ATRTs) are the most common malignant central nervous system tumours in children ≤1 year of age and represent approximately 1–2% of all pediatric brain tumours. ATRT is a primarily monogenic disease characterized by the bi-allelic loss of the SMARCB1 gene, which encodes the hSNF5 subunit of the SWI/SNF chromatin remodeling complex. Though conventional dose chemotherapy is not effective in most ATRT patients, high dose chemotherapy with autologous stem cell transplant, radiotherapy and/or intrathecal chemotherapy all show significant potential to improve patient survival. Recent epigenetic and transcriptional studies highlight three subgroups of ATRT, each with distinct clinical and molecular characteristics with corresponding therapeutic sensitivities, including epigenetic targeting, and inhibition of tyrosine kinases or growth/lineage specific pathways.
