Rhabdoid Tumor

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Retrieved

2022-04-26

Source

Gard

Trials

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Genes

SMARCB1, PGBD5, SMARCA1, SMARCA4, EZH2, CDKN2A, EGFR, BRD9, CCND1, TP53, MDM2, VIM, DNER, PIK3CD, MDM4, STIM1, PLK4, CLDN6, CIC, PROM1, BRD4, PIK3CA, CDKN1A, PIK3CB, PIK3CG, BCOR, MYC, BCR, CDK4, RAB36, TAGLN, SYP, BANF1, WT2, TGFA, VCL, AIFM1, TLE1, ACTB, CROCC, LIN28A, HOTAIR, KRT8P3, MIR206, MIR200C, MIR193A, NUTM1, DMBX1, PHF5A, MIB1, MELK, SLC12A9, FXYD5, DCDC2, RSPH14, CNTNAP2, SMUG1, SPP1, RASSF1, CRTC1, PTK7, SMARCA2, CDKN2B, EWSR1, EGF, E2F1, CTNNB1, CRYBB2, CRX, CLDN4, CNN1, CDKN1C, RPL10, CDK9, CD6, XIAP, BIRC3, BIRC2, ANGPT1, BIN1, ALK, GPC3, HPR, IGF1, IGF2, RPL5, RHD, PTPN11, AKT1, PTGS2, PMAIP1, PLK1, SERPINE1, NSF, NPM1, NFATC2, MYF5, MKI67, MCAM, KRT8, KCNQ1, KCNMA1, DUX4

Drugs

(-)-trans-3-(5,6-dihydro-4H-pyrrolo[3,2,1-ij]quinolin-1yl)-4-(1H-indol-3-yl) pyrrolidine-2, 5-dione, (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate, 16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7-amino acid peptide, 18-(p-(, 4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide, 7-ethyl-10-hydroxy-camptothecin, Anlotinib, Autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4, Autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1, Brostallicin, Camsirubicin, Crenolanib besylate, Deforolimus, Doxorubicin hydrochloride (liposomal) ( DOXIL ), Doxorubicin(6-maleimidocaproyl)hydrazone, Fenretinide, Fibromun, Genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor, Human/murine chimeric monoclonal antibody against endoglin, Larotrectinib ( VITRAKVI ), Mammalian target of rapamcyin (mTOR) inhibitor, N-acetylsarcosyl-glycyl-L-valyl-D-allo-isoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-prolyl-N-ethylamide, Olaratumab ( LARTRUVO ), Ombrabulin, Paclitaxel ( TAXOL ), Palifosfamide, Propranolol hydrochloride ( HEMANGEOL ), Sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein, Trabectedin ( YONDELIS ), Vinorelbine tartrate, Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10

Interested in hearing about new therapies?

Rhabdoid tumor (RT) is an aggressive pediatric soft tissue sarcoma that arises in the kidney, the liver, the peripheral nerves and all miscellaneous soft-parts throughout the body. RT involving the central nervous system (CNS) is called atypical teratoid rhabdoid tumor. RT usually occurs in infancy or childhood. In most cases, the first symptoms are linked to the compressive effects of a bulky tumor (such as respiratory distress, abdominal mass, peripheral nerve palsy). In about 90% of cases it is caused by a mutation in the SMARCB1 gene, which is a tumor suppressor gene. In rare cases, it may be caused by a mutation in the SMARCA4 gene. No standard care exists for RT, although there are many ongoing studies. Treatment includes resection of the tumor mass and chemotherapy and radiotherapy. Because atypical teratoid rhabdoid tumors and rhabdoid tumors of the kidney have the same gene mutation and similar biopsy findings, they are now considered identical or closely related entities. Also, 10-15% of patients with malignant rhabdoid tumors have brain tumors.