Matsuoka Haruki, Yoshida Ken-Ichi, Nakai Sho, Suzuki Rie, Imura Yoshinori, Takami Haruna, Watanabe Makiyo, Wakamatsu Toru, Tamiya Hironari, Outani Hidetatsu, Yagi Toshinari, Kakunaga Shigeki, Takenaka Satoshi
Musculoskeletal Oncology Service, Osaka International Cancer Institute, Osaka 541-8567, Japan.
Department of Orthopaedic Surgery, Osaka Police Hospital, Osaka 543-0035, Japan.
Mol Clin Oncol. 2024 Jul 29;21(4):69. doi: 10.3892/mco.2024.2767. eCollection 2024 Oct.
Undifferentiated pleomorphic sarcoma (UPS) is a high-grade, aggressive soft tissue sarcoma (STS) with a poor prognosis, and no definitive or effective treatment is currently available for it. Pazopanib, an orally available multiple tyrosine kinase inhibitor, has been approved for the treatment of advanced STS. The present study documents the case of a 51-year-old man with advanced UPS with coamplification of platelet-derived growth factor receptor A (), vascular endothelial growth factor receptor 2 () and stem cell factor receptor () genes. The patient exhibited a marked and sustained response to pazopanib. The patient presented with a retroperitoneal tumour with pancreatic head lymph node metastasis, and bone metastases in the second/fifth thoracic vertebrae and left femur. Based on the histological analysis of the retroperitoneal tumour and femoral mass, the patient was diagnosed with UPS. Palliative radiation therapy was administered to the left femur and second/fifth thoracic vertebrae to prevent fractures. After radiation therapy, the patient achieved a partial response after eight courses of doxorubicin. A comprehensive genomic profiling analysis (FoundationOne CDx) revealed coamplification of , and genes. Hence, pazopanib was initiated as a second-line treatment. Notably, the retroperitoneal tumour shrank, and no new lesions developed for 3 years after the initiation of pazopanib treatment. This response suggests that the coamplification of , and may predict favourable outcomes in response to pazopanib.
未分化多形性肉瘤（UPS）是一种高级别、侵袭性软组织肉瘤（STS），预后较差，目前尚无明确或有效的治疗方法。帕唑帕尼是一种口服的多靶点酪氨酸激酶抑制剂，已被批准用于治疗晚期STS。本研究记录了一例51岁患有晚期UPS的男性病例，其血小板衍生生长因子受体A（）、血管内皮生长因子受体2（）和干细胞因子受体（）基因存在共扩增。该患者对帕唑帕尼表现出显著且持续的反应。患者表现为腹膜后肿瘤伴胰头淋巴结转移，以及第二/五胸椎和左股骨骨转移。根据腹膜后肿瘤和股骨肿块的组织学分析，患者被诊断为UPS。对左股骨和第二/五胸椎进行姑息性放射治疗以预防骨折。放射治疗后，患者在接受八个疗程的阿霉素治疗后获得部分缓解。全面基因组图谱分析（FoundationOne CDx）显示、和基因共扩增。因此，开始使用帕唑帕尼作为二线治疗。值得注意的是，腹膜后肿瘤缩小，在开始帕唑帕尼治疗后三年内未出现新病变。这种反应表明、和的共扩增可能预测对帕唑帕尼治疗的良好结果。
