Musculoskeletal Tumor Center, Peking University People's Hospital, No. 11 Xizhimen South Street, Xicheng District, Beijing, P. R. China.
Pathology Department, Peking University People's Hospital, No. 11 Xizhimen South Street, Xicheng District, Beijing, P. R. China.
BMC Cancer. 2024 Feb 3;24(1):172. doi: 10.1186/s12885-024-11921-7.
Epithelioid sarcoma is a rare soft tissue sarcoma characterized by SMARCB1/INI1 deficiency. Much attention has been paid to the selective EZH2 inhibitor tazemetostat, where other systemic treatments are generally ignored. To explore alternative treatment options, we studied the effects of irinotecan-based chemotherapy in a series of epithelioid sarcoma patients.
We retrospectively reviewed data from patients with metastatic or unresectable epithelioid sarcoma at the Peking University People's Hospital treated with irinotecan (50 mg/m/d d Q3W) in combination with Anlotinib (12 mg Qd, 2 weeks on and 1 week off) from July 2015 to November 2021.
A total of 54 courses were administered. With a median follow up of 21.2 months (95% CI, 12.2, 68.1), the 5-year overall survival rate was 83.3%. Five of eight (62.5%) patients presented with unresectable localized lesions, including local tumor thrombosis and lymphatic metastasis. The other patients had unresectable pulmonary metastases. Six of eight (75%) patients had progressed following two lines of systemic therapy. The objective response rate reached 37.5% (three of eight patients) while stabilized disease was observed in 62.5% (five of eight) of patients. No patient had progressed at initial evaluation. At the last follow up, two patients were still using the combination and three patients had ceased the therapy due to toxicities such as diarrhea, nausea, and emesis. One patient changed to tazemetostat for maintenance and one patient stopped treatment due to coronavirus disease 2019 (COVID-19). Another patient stopped therapy as residual lesions had been radiated.
The combination of irinotecan and Anlotinib as a salvage regimen may be considered another effective treatment option for refractory epithelioid sarcoma.
This study was approved in the Medical Ethics Committee of Peking University People's Hospital on October 28, 2022 (No.: 2022PHD015-002). The study was registered in Clinicaltrials.gov with identifier no. NCT05656222.
上皮样肉瘤是一种罕见的软组织肉瘤，其特征是 SMARCB1/INI1 缺失。人们对选择性 EZH2 抑制剂他泽司他（tazemetostat）给予了极大关注，而通常忽略了其他全身治疗方法。为了探索替代治疗方案，我们研究了一系列上皮样肉瘤患者中使用伊立替康为基础的化疗的效果。
我们回顾性分析了 2015 年 7 月至 2021 年 11 月期间在北京大学人民医院接受伊立替康（50mg/m/d d Q3W）联合安罗替尼（12mg Qd，2 周 ON 和 1 周 OFF）治疗的转移性或不可切除的上皮样肉瘤患者的数据。
共给予 54 个疗程。中位随访时间为 21.2 个月（95%CI，12.2，68.1），5 年总生存率为 83.3%。8 例患者中有 5 例（62.5%）为不可切除的局限性病变，包括局部肿瘤血栓形成和淋巴转移。其余患者为不可切除的肺转移。8 例患者中有 6 例（75%）在二线全身治疗后进展。客观缓解率达到 37.5%（8 例患者中有 3 例），62.5%（8 例患者中有 5 例）的患者病情稳定。初始评估时无患者进展。末次随访时，2 例患者仍在使用联合治疗，3 例患者因腹泻、恶心和呕吐等毒性而停止治疗。1 例患者改用他泽司他维持治疗，1 例患者因 2019 年冠状病毒病（COVID-19）而停止治疗。另 1 例患者因残留病灶已接受放疗而停止治疗。
伊立替康联合安罗替尼作为挽救方案可能被认为是治疗难治性上皮样肉瘤的另一种有效治疗选择。
本研究于 2022 年 10 月 28 日获得北京大学人民医院医学伦理委员会批准（编号：2022PHD015-002）。本研究在 Clinicaltrials.gov 注册，标识符为 NCT05656222。
