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000178851 1001_ $$aNemes, Karolina$$b0
000178851 245__ $$aCurrent and Emerging Therapeutic Approaches for Extracranial Malignant Rhabdoid Tumors.
000178851 260__ $$aAlbany, Auckland$$bDove Medical Press$$c2022
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000178851 520__ $$aExtracranial malignant rhabdoid tumors (extracranial MRT) are rare, highly aggressive malignancies affecting mainly infants and children younger than 3 years. Common anatomic sites comprise the kidneys (RTK - rhabdoid tumor of kidney) and other soft tissues (eMRT - extracranial, extrarenal malignant rhabdoid tumor). The genetic origin of these diseases is linked to biallelic pathogenic variants in the genes SMARCB1, or rarely SMARCA4, encoding subunits of the SWI/SNF chromatin-remodeling complex. Even if extracranial MRT seem to be quite homogeneous, recent epigenome analyses reveal a certain degree of epigenetic heterogeneity. Use of intensified therapies has modestly improved survival for extracranial MRT. Patients at standard risk profit from conventional therapies; most high-risk patients still experience a dismal course and often therapy resistance. Discoveries of clinical and molecular hallmarks and the exploration of experimental therapeutic approaches open exciting perspectives for clinical and molecularly stratified experimental treatment approaches. To ultimately improve the outcome of patients with extracranial MRTs, they need to be characterized and stratified clinically and molecularly. High-risk patients need novel therapeutic approaches including selective experimental agents in phase I/II clinical trials.
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000178851 650_7 $$2Other$$aRTK
000178851 650_7 $$2Other$$aeMRT
000178851 650_7 $$2Other$$aexperimental therapy
000178851 650_7 $$2Other$$aextracranial malignant rhabdoid tumors
000178851 650_7 $$2Other$$aimmunotherapy
000178851 7001_ $$0P:(DE-He78)3fdc3623477264cb5d0e14f256dbfbb8$$aJohann, Pascal D$$b1$$udkfz
000178851 7001_ $$aTüchert, Stefanie$$b2
000178851 7001_ $$aMelchior, Patrick$$b3
000178851 7001_ $$aVokuhl, Christian$$b4
000178851 7001_ $$aSiebert, Reiner$$b5
000178851 7001_ $$00000-0002-1967-8343$$aFurtwängler, Rhoikos$$b6
000178851 7001_ $$aFrühwald, Michael C$$b7
000178851 773__ $$0PERI:(DE-600)2508013-1$$a10.2147/CMAR.S289544$$gVol. 14$$p479-498$$tCancer management and research$$v14$$x1179-1322$$y2022
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