Rhabdomyosarcoma

PRAME is an antigen that has been described in primary cutaneous and metastatic melanomas and has become a useful ancillary immunohistochemical marker, particularly when all others are negative. We present a patient with metastatic melanoma to the left atrium with loss of melanocytic differentiation, gain of a rhabdomyosarcomatous differentiation, and a diffuse and strong positivity for PRAME.

This phenomenon was evidenced by the following indicators: reduced clone formation, increased cell apoptosis, cell cycle arrest, as well as the inhibition of tumor cell proliferation. Taken together, our study revealed that shepherdin not only modulates the biological behavior of RMS cells but also enhances their sensitivity to X-ray irradiation, demonstrating its potential as an effective therapeutic strategy for RMS.

This updated review synthesizes current knowledge of DICER1 biology, the historical evolution of the syndrome, the distinctive clinicopathologic features of associated tumors, provides a practical diagnostic algorithm to guide pathologists in recognizing sentinel lesions and initiating germline testing, and recent advances in the study of this syndrome and the DICER1 gene. By integrating molecular mechanisms with evolving clinical practice, this article aims to equip diagnostic pathologists and multidisciplinary teams with diagnostic tools and correlations for the detection and management of DICER1 syndrome.

CART-Vac effectively mediated transient expression, significantly enhancing CAR T expansion and antitumor activity in both models. These findings suggest that CART-Vac can modulate the TME, offering a promising strategy to improve the therapeutic efficacy of CAR T cells in solid tumors.

In conclusion, our multi-omics analysis identifies HMGA2 fusion as a promising diagnostic and prognostic biomarker for DDLPS with rhabdomyosarcomatous differentiation. The comprehensive molecular and immune landscape of this tumor subtype not only deepens our understanding of its pathophysiology but also provides critical insights for future precision medicine strategies.

P1, N=25, Recruiting, University of California, Irvine

The patient received Vincristine, Adriamycin, Cyclophosphamide Ifosfamide, Etoposide (VAC/IE) chemotherapy according to the European Ewing tumour Working Initiative of National Groups (EUROE.W.I.N.G.) protocol and achieved full neurological recovery. This case illustrates the diagnostic and therapeutic challenges posed by Ewing-like sarcomas in the absence of molecular confirmation. Function-preserving subtotal resection combined with multimodal chemotherapy may lead to excellent outcomes and may be preferable to radical resection when the latter carries a high neurological risk.

Clinical translation of these findings as well as conduction of further preclinical research requires an international, multidisciplinary collaborative effort to propel preclinical and clinical studies. This review was conducted by the DSRCT Working Group of OCTOPUS ("Optimising Combination Therapy fOr Paediatric, adolescent and yoUng adult patients with non-rhabdomyosarcoma soft tissue Sarcomas") to consolidate existing knowledge on preclinical translational aspects of DSRCT and guide future cooperative research.

Targeting ROR2 with antibody-drug conjugates kills MYOD1L122R-mutated tumor cells, offering therapeutic opportunities. These findings provide insights into how MYOD1L122R rewires rhabdomyosarcoma to a stem-like state and defines a unique class of oncogenic transcription factors found in aggressive cancers.

Targeted RNA sequencing identified a novel NCOA1::ZNF143 fusion transcript. Despite intensive multimodal treatment including chemotherapy, radiotherapy, surgery, and metronomic therapy, the disease demonstrated multiple relapses with subsequent metastatic progression involving the spinal cord and central nervous system, ultimately leading to a fatal outcome.

Notably, TZM monotherapy inhibited tumor growth in both FN- and FP-RMSCRR xenografts, uncovering a therapy-induced vulnerability. Our integrative multi-omics analysis reveals EZH2-dependent molecular programs underpinning radioresistance and supports EZH2 targeting as a rational radiosensitizing and therapeutic strategy in RMS, including recurrent and RT-refractory disease.

Three cycles of VAC (vincristine + actinomycin D + cyclophosphamide) combination chemotherapy and another cervical conization surgery were taken out for maximum therapeutic effect. This rare case reminds us that the tumor diagnosis should always be taken into consideration for any unexpected mass. A multi-disciplinary team including gynecologist, pediatrician, oncologist, radiologist, and pathologist could be a good choice to avoid such a pitfall of misdiagnosis.