Ewing Sarcoma

Histology and immunohistochemistry (CD99 +, INI-1 +, NKX2.2 +, Ki-67 ≈ 60%) confirmed the diagnosis. This is the first Moroccan and the seventh case of primary hepatic ES reported in the literature, emphasizing the need to consider this entity in the differential diagnosis of pediatric liver tumors.

Together, these results establish vmwhere as a scalable framework for resolving population-level microsatellite variation and linking repeat architecture to chromatin state. Repeat structure and length characteristics provides insights into genotype-function relationships at microsatellite repeats in cancer.

Here we provide the basis for further development of trabectedin/irinotecan for patients with ES by the international cooperative groups. ClinicalTrials.gov: NCT04067115 .

P1, N=48, Recruiting, St. Jude Children's Research Hospital | N=32 --> 48 | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027

P1, N=30, Recruiting, New York Medical College | Trial completion date: Dec 2024 --> Dec 2027 | Trial primary completion date: Dec 2023 --> Dec 2026

Tumors were driven by the col2a1a promoter and resulted in ~70% incidence of notochord tumors within the first 72-96 h. Of the surviving fish, ~5% developed CD99-positive small round blue cell tumors at ~9 months post-fertilization. This work establishes a stable tissue-specific transgenic model of ES, providing a powerful in vivo platform to investigate disease pathogenesis and evaluate novel therapeutic strategies.

P2, N=46, Active, not recruiting, Eli Lilly and Company | Trial completion date: Sep 2028 --> Dec 2026

Drug sensitivity testing of PDCs from bone sarcomas is a valuable tool for a rapid identification of potential treatments, in otherwise genetically complex group of tumors where genome-based assays are difficult to implement. Clones with different phenotypes can outgrow in PDC cultures and can represent clonal evolution that give raise to tumor recurrence.

IL1RAP expression rendered these malignancies similarly vulnerable to IL1RAP-targeting ADCs, which effectively blocked growth of ALCL xenografts and syngeneic ETV6-NTRK3+ sarcomas. Lack of detectable normal tissue toxicity, including in non-human primates, support the further clinical translation of IL1RAP-targeting ADCs.

Postoperatively, she was treated with an Ewing sarcoma‑based chemotherapy protocol (vincristine, doxorubicin, cyclophosphamide/ifosfamide and etoposide (VDC/IE)), but unfortunately, she died 10 months after surgery. While on one hand, among adolescents, fertility preservation poses an additional ethical and therapeutic challenge, on the other hand, rapid, individualised decision‑making is also required to prevent clinical deterioration. This case highlights the diagnostic and therapeutic dilemmas faced, the need to consider PNET in the differential diagnosis of aggressive uterine masses even in adolescents, the important role of ancillary testing like IHC and molecular studies for a definitive diagnosis and the difficulties in balancing life‑saving surgery and fertility preservation when disease is advanced.

The patient exhibited vascular endothelial growth factor receptor amplification, mutations in CHEK1, ERCC3, and TP53, deletion of CD274 (gene of PD-L1), and microsatellite stability. These findings suggest that immunotherapy may be beneficial to URCS patients with low PD-L1 expression and microsatellite stability, when accompanied by immunotherapy-associated genetic alterations.

Targeted and molecular therapies in EWS show significant promise, particularly in rational combinations that exploit the tumor's dependence on EWS-FLI1-driven transcriptional dysregulation, DNA damage repair deficiencies, or survival signaling. Future research must prioritize biomarker-driven patient selection, integration of multiomics approaches, and multicenter prospective trials to translate these strategies into clinically meaningful improvements in EWS survival.