Advancing biology-based therapeutic approaches for atypical teratoid rhabdoid tumors

被引：27

作者：

Hoffman, Lindsey M. ^{[1
]}

Richardson, Elizabeth Anne ^{[2
,3
,4
]}

Ho, Ben ^{[2
,3
,4
]}

Margol, Ashley ^{[5
,6
]}

Reddy, Alyssa ^{[7
,8
]}

Lafay-Cousin, Lucie ^{[9
,10
,11
]}

Chi, Susan ^{[12
,13
,14
]}

Slavc, Irene ^{[15
,16
]}

Judkins, Alexander ^{[17
,18
,19
]}

Hasselblatt, Martin ^{[20
]}

Bourdeaut, Franck ^{[21
,22
,23
,24
]}

Fruhwald, Michael C. ^{[25
,26
,27
]}

Vibhakar, Rajeev ^{[28
,29
,30
]}

Bouffet, Eric ^{[31
,32
,33
]}

Huang, Annie ^{[2
,3
,4
,32
,33
,34
]}

机构：

[1] Phoenix Childrens Hosp, Ctr Canc & Blood Disorders, Phoenix, AZ USA

[2] Hosp Sick Children, Arthur & Sonia Labatt Brain Tumour Res Ctr, Toronto, ON, Canada

[3] Hosp Sick Children, Dept Cell Biol, Toronto, ON, Canada

[4] Univ Toronto, Fac Med, Dept Lab Med & Pathobiol, Toronto, ON, Canada

[5] Childrens Hosp Los Angeles, Canc & Blood Dis Inst, Los Angeles, CA 90027 USA

[6] Univ Southern Calif, Dept Pediat, Keck Sch Med, Los Angeles, CA 90007 USA

[7] Univ Calif San Francisco, Dept Neurol, San Francisco, CA 94143 USA

[8] Univ Calif San Francisco, Dept Pediat, San Francisco, CA 94143 USA

[9] Alberta Childrens Prov Gen Hosp, Dept Pediat Hematol Oncol & Blood & Marrow Transp, Calgary, AB, Canada

[10] Univ Calgary, Dept Paediat, Cumming Sch Med, Calgary, AB, Canada

[11] Univ Calgary, Dept Oncol, Cumming Sch Med, Calgary, AB, Canada

[12] Dana Farber Canc Inst, Pediat Med Neurooncol, Boston, MA 02115 USA

[13] Dana Farber Boston Childrens Canc & Blood Disorde, Boston, MA USA

[14] Harvard Med Sch, Dept Pediat, Boston, MA 02115 USA

[15] Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria

[16] Med Univ Vienna, Dept Pediat & Adolescent Med, Div Neonatol Pediat Intens Care & Neuropediat, Vienna, Austria

[17] Childrens Hosp Los Angeles, Ctr Personalized Med, Los Angeles, CA 90027 USA

[18] Childrens Hosp Los Angeles, Pathol & Lab Med, Los Angeles, CA 90027 USA

[19] Univ Southern Calif, Dept Pathol, Keck Sch Med, Los Angeles, CA 90007 USA

[20] Univ Hosp Munster, Inst Neuropathol, Munster, Germany

[21] Curie Inst, Integrated Canc Res Site, Paris, France

[22] Curie Inst, Dept Genet, Paris, France

[23] Curie Inst, Dept Oncopediatry & Young Adults, Paris, France

[24] Curie Inst, INSERM U830, Lab Translat Res Pediat Oncol, SIREDO Pediat Oncol Ctr, Paris, France

[25] Univ Hosp Augsburg, Univ Childrens Hosp, Swabian Childrens Canc Ctr, Augsburg, Germany

[26] Univ Munster, Univ Childrens Hosp Munster, Dept Pediat Hematol & Oncol, Munster, Germany

[27] EU RHAB Registry Working Grp, Augsburg, Germany

[28] Univ Colorado Denver, Dept Pediat, Anschutz Med Campus, Aurora, CO USA

[29] Childrens Hosp Colorado, Morgan Adams Fdn, Pediat Brain Tumor Res Program, Aurora, CO USA

[30] Univ Colorado Denver, Dept Neurosurg, Aurora, CO USA

[31] SickKids Res Inst, Child Hlth Evaluat Sci, Toronto, ON, Canada

[32] Univ Toronto, Dept Paediat, Toronto, ON, Canada

[33] Hosp Sick Children, Div Hematol Oncol, 555 Univ Ave, Toronto, ON, Canada

[34] Univ Toronto, Fac Med, Dept Med Biophys, Toronto, ON, Canada

来源：

NEURO-ONCOLOGY | 2020年 / 22卷 / 07期

关键词：

ATRT; enhancer; epigenomics; rhabdoid tumors; subgroup-specific therapeutics; CENTRAL-NERVOUS-SYSTEM; HIGH-DOSE CHEMOTHERAPY; PRIMITIVE NEUROECTODERMAL TUMORS; STANDARD ANTICANCER AGENTS; PROGENITOR-CELL RESCUE; TERATOID/RHABDOID TUMORS; BRAIN-TUMORS; PEDIATRIC-PATIENTS; YOUNG-CHILDREN; PHASE-I;

D O I：

10.1093/neuonc/noaa046

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Atypical teratoid rhabdoid tumor (ATRT) is a rare, highly malignant central nervous system cancer arising in infants and younger children, historically considered to be homogeneous, monogenic, and incurable. Recent use of intensified therapies has modestly improved survival for ATRT, however, a majority of patients will still succumb to their disease. While ATRTs almost universally exhibit loss of SMARCB1 (BAF47/INI1/SNF5), recent whole genome, transcriptome, and epigenomic analyses of large cohorts reveal previously underappreciated molecular heterogeneity. These discoveries provide novel insights into how SMARCB1 loss drives oncogenesis and confer specific therapeutic vulnerabilities, raising exciting prospects for molecularly stratified treatment for patients with ATRT.

引用

页码：944 / 954

页数：11

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