Li Yong, Huang Jinpeng, Chen Xian, Ye Yongsong, Du Xiaohua, Voutsadakis Ioannis A, Seetharam Mahesh, Zhang Haibo, Lu Min
Department of Oncology, The Second Affiliated Hospital of Guangzhou University of Chinese Medicine, Guangzhou, China.
Department of Oncology, Guangdong Provincial Hospital of Traditional Chinese Medicine, Guangzhou, China.
Front Oncol. 2024 Jul 11;14:1416241. doi: 10.3389/fonc.2024.1416241. eCollection 2024.
Undifferentiated small round-cell sarcomas (uSRCSs) are a subgroup of sarcomas that are difficult to diagnose. Some uSRCSs have specific gene re-arrangements, but others do not. Currently, there is no specific treatments for advanced uSRCSs, and its treatment is largely based on general experience with sarcomas, which includes chemotherapy, targeted therapy, and immunotherapy. In this article, we report a patient with uSRCS who responded to treatment with anti-VEGF inhibitor surufatinib and anti-PD-1 inhibitor camrelizumab after progression on first-line chemotherapy, second-line anlotinib combined with immunotherapy, and third-line chemotherapy.
In July 2020, a 37-year-old female patient was diagnosed with advanced uSRCS. Results for the Ewing sarcoma RNA binding protein 1 and Wilms tumor suppressor (EWSR1/WT1) fusion gene were negative. The patient was also negative with BCOR (BCL6 co-repressor) and CIC (capicua transcriptional repressor) fusion gene. The next-generation sequencing results revealed point mutations on Phosphatidylinositol-4,5-Bisphosphate 3-Kinase Catalytic Subunit Beta (PIK3CB), Transcription Factor Binding To IGHM Enhancer 3 (TFE3), Mucin 16 (MUC16), and AXL (Axl, also called UFO, ARK, and Tyro7, is part of a family of receptor tyrosine kinases). The patient received 4 cycles of the Ifosfamide and epirubicin hydrochloride regimen, and her best objective response was stable disease. On November 3, 2020, a computed tomography (CT) scan revealed progressive disease (PD). Two cycles of camrelizumab (a programmed death-1 inhibitor) plus anlotinib (an anti- vascular endothelial growth factor drug) were administered, but PD was again observed. Thus, a regimen of gemcitabine plus docetaxel was adopted. Unfortunately, the disease progressed once again after two cycles of the treatment. On February 4, 2021, the patient began to receive targeted therapy with surufatinib combined with camrelizumab. A CT scan showed that the tumor achieved a partial response. As of April 2023, the patient had a progression-free survival time of 26 months.
Surufatinib in combination with camrelizumab could be effective in the treatment of advanced uSRCSs.
未分化小圆细胞肉瘤（uSRCSs）是一类难以诊断的肉瘤亚型。部分uSRCSs存在特定的基因重排，但其他的则没有。目前，晚期uSRCSs尚无特异性治疗方法，其治疗主要基于肉瘤的一般经验，包括化疗、靶向治疗和免疫治疗。在本文中，我们报告了1例uSRCS患者，该患者在一线化疗、二线安罗替尼联合免疫治疗及三线化疗进展后，对抗血管内皮生长因子（VEGF）抑制剂苏尼替尼和抗程序性死亡蛋白1（PD-1）抑制剂卡瑞利珠单抗治疗有反应。
2020年7月，一名37岁女性患者被诊断为晚期uSRCS。尤因肉瘤RNA结合蛋白1和威尔姆斯肿瘤抑制因子（EWSR1/WT1）融合基因检测结果为阴性。该患者的BCOR（BCL6共抑制因子）和CIC（capicua转录抑制因子）融合基因检测结果也为阴性。二代测序结果显示磷脂酰肌醇-4,5-二磷酸3-激酶催化亚基β（PIK3CB）、转录因子结合IGHM增强子3（TFE3）、粘蛋白16（MUC16）和AXL（Axl，也称为UFO、ARK和Tyro7，是受体酪氨酸激酶家族的一部分）存在点突变。患者接受了4周期异环磷酰胺和盐酸表柔比星方案治疗，其最佳客观反应为疾病稳定。2020年11月3日，计算机断层扫描（CT）显示疾病进展（PD）。给予2周期卡瑞利珠单抗（一种程序性死亡-1抑制剂）加安罗替尼（一种抗血管内皮生长因子药物）治疗，但再次观察到PD。因此，采用吉西他滨加多西他赛方案。不幸的是，两周期治疗后疾病再次进展。2021年2月4日，患者开始接受苏尼替尼联合卡瑞利珠单抗的靶向治疗。CT扫描显示肿瘤获得部分缓解。截至2023年4月，患者的无进展生存期为26个月。
苏尼替尼联合卡瑞利珠单抗可能对晚期uSRCSs治疗有效。
