Biology and Treatment of Rhabdoid Tumor.
Rhabdoid tumor is a rare, highly aggressive malignancy that primarily affects infants and young children. These tumors typically arise in the brain and kidney, although extrarenal, non-central nervous system tumors in almost all soft-tissue sites have been described. SMARCB1 is a member of the SWI/SNF chromatin-remodeling complex and functions as a tumor suppressor in the vast majority of rhabdoid tumors. Patients with germline mutations or deletions affecting SMARCB1 are predisposed to the development of rhabdoid tumors, as well as the genetic disorder schwannomatosis. The current hypothesis is that rhabdoid tumors are driven by epigenetic dysregulation, as opposed to the alteration of a specific biologic pathway. The strategies for novel therapeutic approaches based on what is currently known about rhabdoid tumor biology are presented.
Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
James I
JI
Geller
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Jacquelyn J
JJ
Roth
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles; Keck School of Medicine, University of Southern California, Los Angeles, Ca.
Jaclyn A
JA
Biegel
D000818:Animals; D002868:Chromosomal Proteins, Non-Histone / Q000235:genetics*; D004268:DNA-Binding Proteins / Q000235:genetics*; D044127:Epigenesis, Genetic / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods / Q000639:trends; D006801:Humans; D018335:Rhabdoid Tumor / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D000071796:SMARCB1 Protein; D014157:Transcription Factors / Q000235:genetics*; D016896:Treatment Outcome
D002868:Chromosomal Proteins, Non-Histone; D004268:DNA-Binding Proteins; D000071796:SMARCB1 Protein; C513266:SMARCB1 protein, human; D014157:Transcription Factors