Rhabdomyosarcoma

However, these frequency estimates are derived from a subset of tested cases and should be interpreted within the context of variable molecular data reporting across studies. These findings highlight the potential value molecularly guided treatment strategies in this rare and lethal disease subtype.

The treatment protocol and adjuvant drugs differ significantly from prostatic adenocarcinoma or small cell carcinoma, necessitating an accurate histological diagnosis for appropriate patient management. A molecular work-up can be useful in challenging specimens.

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

After a dose of ifosfamide/etoposide elsewhere, therapy was revised to ifosfamide, carboplatin, and etoposide plus brentuximab vedotin, with marked improvement by 2 cycles and cutaneous resolution by 5; she died of cardiopulmonary failure before the sixth cycle. This case underscores generous sampling, focused immunohistochemistry, and staging; negative ALK/EMA does not exclude systemic disease, and CD30-directed therapy can yield rapid cutaneous responses.

No additional adjuvant treatment was given, and the patient is alive without disease, 8 months after the major resection. Long term follow-up of 6 years with only local recurrence lends further support to the notion that these neoplasms are a class of indolent/low-grade rhabdomyoblastic tumors that are biologically and clinically distinct from fully malignant spindle cell rhabdomyosarcomas.

The Riyadh congress reaffirmed that survival disparities in Asia are driven by addressable inequities. By advancing diagnostics, medicines, registries, and collaborative trials, SIOP Asia and its partners demonstrated a pathway toward narrowing the survival gap.

The patient was treated with four cycles of paclitaxel and carboplatin, followed by an additional four cycles of combination chemotherapy with durvalumab...Maintenance therapy with durvalumab and olaparib was initiated, and the patient has remained progression-free for 10 months...The case highlights the value of genomic profiling and germline testing for personalized treatment strategies. The successful use of platinum-based chemotherapy, immune checkpoint blockade and poly (ADP-ribose) polymerase inhibition to treat this HRD-positive, mismatch repair-proficient tumor demonstrates the potential of biomarker-driven therapy for UCS.

P=N/A, N=143, Not yet recruiting, Dana-Farber Cancer Institute

Our results also demonstrate that inducible knockdown of TAK1 in human RMS xenografts retards tumor growth and enhances myogenic differentiation in vivo. Collectively, these findings uncover a previously unrecognized role for TAK1 in RMS growth and differentiation, and suggest that TAK1 can be a potential therapeutic target for the treatment of RMS.

Pediatric maxillofacial RMS represents a distinct subgroup with a prognosis intermediate to that of other head and neck subsites. Outcomes are heavily influenced by early chemotherapy response, as well as tumor biology.

P=N/A, N=100, Not yet recruiting, St. Jude Children's Research Hospital