TY  - JOUR
AU  - Nemes, Karolina
AU  - Johann, Pascal D
AU  - Tüchert, Stefanie
AU  - Melchior, Patrick
AU  - Vokuhl, Christian
AU  - Siebert, Reiner
AU  - Furtwängler, Rhoikos
AU  - Frühwald, Michael C
TI  - Current and Emerging Therapeutic Approaches for Extracranial Malignant Rhabdoid Tumors.
JO  - Cancer management and research
VL  - 14
SN  - 1179-1322
CY  - Albany, Auckland
PB  - Dove Medical Press
M1  - DKFZ-2022-00307
SP  - 479-498
PY  - 2022
AB  - Extracranial malignant rhabdoid tumors (extracranial MRT) are rare, highly aggressive malignancies affecting mainly infants and children younger than 3 years. Common anatomic sites comprise the kidneys (RTK - rhabdoid tumor of kidney) and other soft tissues (eMRT - extracranial, extrarenal malignant rhabdoid tumor). The genetic origin of these diseases is linked to biallelic pathogenic variants in the genes SMARCB1, or rarely SMARCA4, encoding subunits of the SWI/SNF chromatin-remodeling complex. Even if extracranial MRT seem to be quite homogeneous, recent epigenome analyses reveal a certain degree of epigenetic heterogeneity. Use of intensified therapies has modestly improved survival for extracranial MRT. Patients at standard risk profit from conventional therapies; most high-risk patients still experience a dismal course and often therapy resistance. Discoveries of clinical and molecular hallmarks and the exploration of experimental therapeutic approaches open exciting perspectives for clinical and molecularly stratified experimental treatment approaches. To ultimately improve the outcome of patients with extracranial MRTs, they need to be characterized and stratified clinically and molecularly. High-risk patients need novel therapeutic approaches including selective experimental agents in phase I/II clinical trials.
KW  - RTK (Other)
KW  - eMRT (Other)
KW  - experimental therapy (Other)
KW  - extracranial malignant rhabdoid tumors (Other)
KW  - immunotherapy (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:35173482
C2  - pmc:PMC8841298
DO  - DOI:10.2147/CMAR.S289544
UR  - https://inrepo02.dkfz.de/record/178851
ER  -