BACKGROUND: Rhabdomyosarcoma (RMS) is one of the most common soft tissue sarcomas in children and adolescents, in which PAX3-FOXO1 fusion gene positive patients have very poor prognosis. PAX3-FOXO1 has been identified as an independent prognostic predictor in RMS, with no currently available targeted therapeutic intervention. The novel tyrosine kinase inhibitor anlotinib exhibits a wide range of anticancer effects in multiple types of cancers; however, there have been no relevant studies regarding its application in RMS.
METHODS: We investigated the effects of PAX3-FOXO1 on the therapeutic efficacy of anlotinib using the CCK-8 assay, flow cytometry, invasion assay, wound healing assay, western blotting, quantitative polymerase chain reaction(qPCR), and xenograft experiments. RNA-seq and co-immunoprecipitation assays were conducted to determine the specific mechanism by which anlotinib regulates PAX3-FOXO1 expression.
RESULTS: Anlotinib effectively inhibited RMS cell proliferation and promoted apoptosis and G2/M phase arrest while impeding tumor growth in vivo. Downregulation of PAX3-FOXO1 enhances the antitumor effects of anlotinib. Anlotinib upregulates protein kinase NEK2 and increases the degradation of PAX3-FOXO1 via the ubiquitin-proteasome pathway, leading to a reduction in PAX3-FOXO1 protein levels.
CONCLUSIONS: Anlotinib effectively inhibited the malignant progression of RMS and promoted degradation of the fusion protein PAX3-FOXO1. Anlotinib could be a targeted therapeutic approach to treat PAX3-FOXO1 fusion-positive RMS.

The management of pediatric tumors is complex, with surgery, chemotherapy, and radiotherapy being cornerstones in their treatment. Tumor removal is increasingly performed by a minimally invasive approach, which allows for quicker postoperative recovery and less postoperative pain. The goal of this report is to give an overview of minimally invasive surgical approaches for common pediatric tumors, with a focus on technical considerations and postoperative outcomes.

BACKGROUND: Despite a multimodal approach including surgery, chemo- and radiotherapy, the 5-year event-free survival rate for rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in childhood, remains very poor for metastatic patients, mainly due to the selection and proliferation of tumour cells driving resistance mechanisms. Personalised medicine-based protocols using new drugs or targeted therapies in combination with conventional treatments have the potential to enhance the therapeutic effects, while minimizing damage to healthy tissues in a wide range of human malignancies, with several clinical trials being started. In this study, we analysed, for the first time, the antitumour activity of SFX-01, a complex of synthetic d, l-sulforaphane stabilised in alpha-cyclodextrin (Evgen Pharma plc, UK), used as single agent and in combination with irradiation, in four preclinical models of alveolar and embryonal RMS. Indeed, SFX-01 has shown promise in preclinical studies for its ability to modulate cellular pathways involved in inflammation and oxidative stress that are essential to be controlled in cancer treatment.
METHODS: RH30, RH4 (alveolar RMS), RD and JR1 (embryonal RMS) cell lines as well as mouse xenograft models of RMS were used to evaluate the biological and molecular effects induced by SFX-01 treatment. Flow cytometry and the modulation of key markers analysed by q-PCR and Western blot were used to assess cell proliferation, apoptosis, autophagy and production of intracellular reactive oxygen species (ROS) in RMS cells exposed to SFX-01. The ability to migrate and invade was also investigated with specific assays. The possible synergistic effects between SFX-01 and ionising radiation (IR) was studied in both the in vitro and in vivo studies. Student\'s t-test or two-way ANOVA were used to test the statistical significance of two or more comparisons, respectively.
RESULTS: SFX-01 treatment exhibited cytostatic and cytotoxic effects, mediated by G2 cell cycle arrest, apoptosis induction and suppression of autophagy. Moreover, SFX-01 was able to inhibit the formation and the proliferation of 3D tumorspheres as monotherapy and in combination with IR. Finally, SFX-01, when orally administered as single agent, displayed a pattern of efficacy at reducing the growth of tumour masses in RMS xenograft mouse models; when combined with a radiotherapy regime, it was observed to act synergistically, resulting in a more positive outcome than would be expected by adding each exposure alone.
CONCLUSIONS: In summary, our results provide evidence for the antitumour properties of SFX-01 in preclinical models of RMS tumours, both as a standalone treatment and in combination with irradiation. These forthcoming findings are crucial for deeper investigations of SFX-01 molecular mechanisms against RMS and for setting up clinical trials in RMS patients in order to use the SFX-01/IR co-treatment as a promising therapeutic approach, particularly in the clinical management of aggressive RMS disease.

Rhabdomyosarcoma is a common soft tissue tumor in children but rare in adults. Alveolar rhabdomyosarcoma represents a subtype of rhabdomyosarcoma, extremely rare in adults, especially within the nasal cavities. Therapeutic protocols for adults are often based on those used in pediatric cases. We present the case of a 56-year-old female patient with a history of breast cancer who developed alveolar rhabdomyosarcoma of the nasal cavity, stage III, managed initially with chemotherapy resulting in partial response. Subsequently, the patient underwent concomitant chemoradiotherapy. The clinical course was marked by local remission with metastatic progression after 18 months. Alveolar rhabdomyosarcoma is uncommon in adults, and its therapeutic management remains non-standardized. However, it is typically based on initial chemotherapy followed by local treatment. Despite therapeutic advances, the prognosis remains poor.

Objective:To explore the imaging features of rare tumors of nasal cavity and sinuses, and to improve the understanding of these diseases, thereby aiding clinical diagnosis and treatment. Methods:The CT and MRI findings of 79 cases of rare neoplasm of nasal cavity and sinuses confirmed by pathology were retrospectively analyzed, and the imaging features were summarized. Results:Among the 79 cases, there were 16 cases of neuroendocrine carcinoma, most showing expansive and infiltrative bone destruction without hyperosteogeny and sclerosis. The sphenoid sinus exhibited a \"pigeon\" shape. In 28 cases of malignant melanoma, MRI signals were diverse, typical signals were rare, but mixed signals were more common. In 12 cases of rhabdomyosarcoma, MRI enhancement mostly showed \"grape-like\" enhancement and partial ring enhancement; There were 10 cases of olfactory neuroblastoma, the lesions were consistent with the distribution area of olfactory mucosa, most of them were lobulated, marginal nodules, and \"flower ring\" enhancement, and 2 cases grew across intracranial and external, with multiple cystic lesions and surrounding flaky edema bands. In 5 cases of solitary fibrous tumor, Benign tumors had regular shape and uniform density, while malignant tumors had irregular shape and uneven density, The enhancement was obviously uneven and showed a \"pattern\" change. There were 2 cases of sarcomatoid carcinoma, both with lobed appearance, uneven density, lamellar low-density shadow, and osteolytic bone destruction. In 4 cases of schwannoma, the enhancement showed obvious inhomogeneous enhancement. One case showed cystic necrosis, one case showed calcification, and the surrounding structure was compressed without damage. There was 1 case of neurofibroma, with many cystic components, low signal separation and compartmentalized enhancement. One case of paraganglioma showed moderate enhancement in the arterial phase and progressive enhancement in the venous phase, accompanied by significant swelling bone destruction. Conclusion:Rare tumors of nasal cavity and paranasal sinuses have distinctive imaging features. CT and MRI can effectively show the extent of the lesions and the degree of infiltration into adjacent tissues and organs, which is helpful for early clinical diagnosis and staging. However, definitive diagnosis still depends on pathology and immunohistochemistry.
目的：探讨鼻腔鼻窦少见肿瘤的影像学特点，提高对该类疾病的认识，为临床诊断和治疗提供帮助。 方法：回顾性分析经病理证实的79例鼻腔鼻窦少见肿瘤的CT和MRI表现，总结其影像学特征。 结果：79例中，神经内分泌癌16例，骨质破坏多表现为膨胀性骨质破坏与浸润性骨质破坏并存，而不伴骨质增生硬化，位于蝶窦者双侧对称呈“鸽”形；恶性黑色素瘤28例，MRI信号表现多样，典型者少见，而以混杂信号多见；横纹肌肉瘤12例，MRI增强多呈“葡萄状”强化、部分环状强化；嗅神经母细胞瘤10例，病灶与嗅黏膜分布区一致，多呈分叶状，边缘结节状，“花环状”强化，2例跨颅内外生长，颅内病灶见多发囊变及周围片状水肿带；孤立性纤维性肿瘤5例，良性者形态规则，密度均匀，恶性者形态不规则，密度不均，增强明显不均匀强化，呈“地图样”改变；肉瘤样癌2例，形态似分叶，密度不均，内见片状低密度影，均有溶骨性骨质破坏；神经鞘瘤4例，增强呈明显欠均匀强化，1例见囊变坏死，1例见钙化，周围结构受压而无破坏；神经纤维瘤1例，囊变成分多，内见低信号分隔，增强呈分隔样强化；副神经节瘤1例，增强动脉期中度强化，静脉期进行性明显强化，伴有明显膨胀性骨质破坏。 结论：鼻腔鼻窦少见肿瘤具有一定的影像学特征，CT和MRI能更好地显示病灶范围及对邻近组织器官的浸润程度，有助于临床早期诊断和分期，确诊仍需依靠病理和免疫组织化学。.

A geriatric Holland Lop rabbit presented for acute lameness. A pathologic fracture of the right distal femur associated with a pleomorphic rhabdomyosarcoma was diagnosed, and staging radiographs showed no overt metastasis upon initial presentation. The limb was amputated and submitted for microscopic examination. Immunohistochemical evaluation revealed the neoplastic cells were positive for desmin and MyoD1, and negative for cytokeratin AE1/AE3, CD204, IBA-1, and SMA. Gross, histologic, and immunohistochemical evaluation confirmed a diagnosis of pleomorphic rhabdomyosarcoma. The patient died 396 days after amputation, and a post-mortem examination showed metastatic sarcoma to multiple organs.

BACKGROUND: Adult Head and neck Rhabdomyosarcomas (HNRMS) are exceedingly rare and remain challenging for pathologists.
METHODS: Five cases of adult HNRMS (≥19 years) were retrieved from the archives of the Department of Pathology of Hospital of Specialities in Rabat (HSR) in Morocco, over 5 years. Clinical and pathologic findings from hematoxylin and eosin slides and immunohistochemistry for Desmin and Myogenin were reviewed.
UNASSIGNED: The median age was 33, with a men\'s predominance (3 M/2F). Histological analysis revealed three cases of Alveolar Rhabdomyosarcoma (RMS), one Pleomorphic RMS, and one spindle cell/sclerosing RMS. In addition to the typical histology observed in each RMS, we found tricky growth patterns that could be a source of misdiagnosis. All five cases demonstrated variable positivity for Desmin and Myogenin.
CONCLUSIONS: HNRMS cases have different pathological features than pediatric RMS cases. We identified rare subtypes such as pleomorphic and spindle cell/sclerotic RMS, which exhibit unusual morphological patterns.

暂无摘要。

暂无摘要。

Clinical trials conducted by the Intergroup Rhabdomyosarcoma (RMS) Study Group and the Children\'s Oncology Group have been pivotal to establishing current standards for diagnosis and therapy for RMS. Recent advancements in understanding the biology and clinical behavior of RMS have led to more nuanced approaches to diagnosis, risk stratification, and treatment. The complexities introduced by these advancements, coupled with the rarity of RMS, pose challenges to conducting large-scale phase 3 clinical trials to evaluate new treatment strategies for RMS. Given these challenges, systematic planning of future clinical trials in RMS is paramount to address pertinent questions regarding the therapeutic efficacy of drugs, biomarkers of response, treatment-related toxicity, and patient quality of life. Herein, the authors outline the proposed strategic approach of the Children\'s Oncology Group Soft Tissue Sarcoma Committee to the next generation of RMS clinical trials, focusing on five themes: improved novel agent identification and preclinical to clinical translation, more efficient trial development and implementation, expanded opportunities for knowledge generation during trials, therapeutic toxicity reduction and quality of life, and patient engagement.