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抗血管生成药物治疗促结缔组织增生性小圆细胞肿瘤的临床疗效与安全性研究

Clinical efficacy and safety Study of anti-angiogenic drugs in the treatment of desmoplastic small round cell tumors

投稿时间：2026-01-21 修订日期：2026-03-22

DOI：

中文关键词: 促结缔组织增生性小圆细胞肿瘤抗血管生成药物生存分析回顾性研究

英文关键词:desmoplastic small round cell tumors anti-angiogenic drugs survival analysis retrospective study

基金项目:国家自然科学基金项目（面上项目，重点项目，重大项目）；武汉大学“临床医学+X 青年拔尖项目”

作者	单位	邮编
展雨洁	武汉大学人民医院肿瘤中心	430060
陈欣祎	武汉大学人民医院肿瘤中心
金瑶	武汉大学人民医院肿瘤中心
孙锐	武汉大学人民医院肿瘤中心
袁静萍	武汉大学人民医院病理科
彭敏^*	武汉大学人民医院肿瘤中心	430060

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中文摘要:

目的：评估抗血管生成药物在晚期促结缔组织增生性小圆细胞肿瘤（Desmoplastic Small Round Cell Tumor，DSRCT）患者中的临床疗效及安全性。方法：回顾性分析 2021 年 1 月 1 日至 2024 年 12 月 31 日在我院肿瘤中心治疗或随访的 28 例晚期 DSRCT 患者。根据是否接受抗血管生成药物治疗分为治疗组（n=16）和对照组（n=12）。比较两组患者的客观缓解率（Objective Response Rate，ORR）、疾病控制率（Disease?Control?Rate，DCR）、无进展生存期（Progression-Free?Survival，PFS）及治疗相关不良反应，并进行生存分析和预后因素分析。结果：随访截止时，共 17 例患者出现疾病进展。全组患者的中位 PFS 为 12.9 个月（95% CI：5.4～20.3）。接受抗血管生成药物治疗的患者 PFS 显著优于未接受者（18.2个月vs. 6.7个月，P=0.004）。单因素分析显示，手术治疗、是否发生转移及是否存在肝转移与 PFS 显著相关（P＜0.05）。多因素 Cox 回归分析表明，治疗模式是影响 PFS 的独立预后因素。治疗组 ORR 为 62.5%，对照组为 50.0%，差异无统计学意义；治疗组 DCR 显著高于对照组，差异具有统计学意义。（93.8% vs 58.3%，P=0.024）。亚组分析显示，不同抗血管生成药物（安罗替尼、阿帕替尼、贝伐珠单抗）及其使用时机对 PFS 的影响差异均无统计学意义（P＞0.05）。安全性分析显示，两组不良反应总体可控，治疗组高血压发生率较高（31.3% vs. 0%，P = 0.033），未观察到治疗相关死亡。结论：在本研究队列中，抗血管生成药物治疗与晚期 DSRCT 患者更长的无进展生存期相关，且安全性可控，提示其在晚期 DSRCT 治疗中具有潜在临床价值。

英文摘要:

Objective To evaluate the clinical efficacy, safety, and prognostic impact of anti-angiogenic therapy in patients with advanced desmoplastic small round cell tumor (DSRCT). Methods This retrospective study included 28 patients with advanced DSRCT treated or followed up at our institution between January 1, 2021 and December 31, 2024. Patients were divided into a treatment group (n=16) receiving anti-angiogenic agents and a control group (n=12) without anti-angiogenic therapy. Objective response rate (ORR), disease control rate (DCR), progression-free survival (PFS), and treatment-related adverse events were analyzed. Survival analyses and prognostic factor assessments were performed. Results At the time of analysis, disease progression had occurred in 17 patients. The median PFS for the entire cohort was 12.9 months (95% CI: 5.4–20.3). Patients treated with anti-angiogenic therapy achieved significantly longer PFS than those who did not receive such treatment(18.2months vs. 6.7months，P=0.004). Univariate analysis showed that surgery, metastatic status, and liver metastasis were significantly associated with PFS (P<0.05). Multivariate Cox regression analysis identified treatment pattern as an independent prognostic factor for PFS. The ORR was 62.5% in the treatment group and 50.0% in the control group, with no statistically significant difference, whereas the DCR was significantly higher in the treatment group than in the control group (93.8% vs. 58.3%, P=0.024). Subgroup analyses demonstrated no significant differences in PFS according to the type of anti-angiogenic agent (anlotinib, apatinib, or bevacizumab) or the timing of anti-angiogenic therapy (P>0.05). Safety analysis indicated that treatment-related adverse events were generally manageable; hypertension was more frequent in the treatment group (31.3% vs. 0%，P = 0.033), and no treatment-related deaths were observed. Conclusion In this retrospective cohort, anti-angiogenic therapy was associated with prolonged progression-free survival and acceptable safety in patients with advanced DSRCT, suggesting its potential clinical value in this rare and aggressive malignancy.

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