J Clin Invest.
ET-743–mediated adipocytic differentiation in MRCLS patient 1.
(A) Survival curve of ET-743–treated patients by major subtype. LMS, leiomyosarcoma; HGUPS, high-grade undifferentiated pleomorphic sarcoma; DDLS, dedifferentiated liposarcoma; Other, synovial sarcoma, osteosarcoma, or alveolar soft parts sarcoma (see Table 1). P = 0.0572, χ2 distribution, 4 degrees of freedom. (B) Histopathology of MRCLS patient 1 before (week 1) and after 2 rounds of (week 6) ET-743 treatment. Original magnification, ×100. (C) CT imaging of MRCLS patient 1 before and after ET-743, as well as 2 years after discontinuing ET-743 (week 158). (D and E) IHC of p53 in MRCLS patient 1 before (D) and after (E) ET-743 treatment. Mutational analysis revealed a TP53 mutation (exon 7, codon 225, Val>Ala), in this patient. (F–K) IHC of the 6 other MRCLS patients in our series, none of which displayed a mutation (Supplemental Table 2). Original magnification, ×200.