| 展雨洁,陈欣祎,金 瑶,等.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[J].肿瘤学杂志,2026,32(5):391-397. |
| 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 of Anti-Angiogenic Drugs in the Treatment of Desmoplastic Small Round Cell Tumors |
| 投稿时间：2026-01-21 |
| DOI：10.11735/j.issn.1671-170X.2026.05.B006 |
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| 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 疗效 生存 |
| 英文关键词:desmoplastic small round cell tumors anti-angiogenic drugs efficacy survival |
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| 中文摘要: |
| 摘 要：［目的］ 评估抗血管生成药物在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日在武汉大学人民医院肿瘤中心治疗或随访的28例Ⅲ~Ⅳ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］ 随访截止至2024年12月31日，共17例患者出现疾病进展。治疗组ORR为62.5%，对照组为50.0%，差异无统计学意义（χ2=0.438，P=0.508）；治疗组DCR高于对照组，差异具有统计学意义（93.8% vs 58.3%，χ2=5.109，P=0.024）。全组患者中位PFS为12.9个月（95%CI：5.4～20.3个月）。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、远处转移及肝转移均与PFS显著性相关（P均＜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（HR=5.228，95%CI：1.357~20.145）。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均＞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），两组均未观察到治疗相关死亡。［结论］ 抗血管生成药物治疗与晚期 DSRCT 患者更长PFS相关，且安全性可控，提示抗血管生成药物在晚期 DSRCT 治疗中具有潜在临床价值。 |
| 英文摘要: |
| Abstract：［Objective］ To evaluate the clinical efficacy and safety of anti-angiogenic drugs in patients with desmoplastic small round cell tumor (DSRCT). ［Methods］ A retrospective analysis was conducted on 28 patients with stage Ⅲ~Ⅳ DSRCT treated or followed up at the Cancer Center of Renmin Hospital of Wuhan University from January 1， 2021 to December 31， 2024. They were divided into a treatment group (received anti-angiogenic drugs， n=16) and a control group (did not receive anti-angiogenic drugs， n=12). The objective response rate (ORR)， disease control rate (DCR)， progression-free survival (PFS) and treatment-related adverse events were compared between the two groups. Survival analysis and prognostic factor analysis were performed. ［Results］ By the follow-up cutoff date of December 31， 2024， 17 patients had experienced disease progression. The ORR was 62.5% in the treatment group and 50.0% in the control group (χ2=0.438， P=0.508). The DCR in the treatment group was significantly higher than that in the control group (93.8% vs 58.3%， χ2=5.109， P=0.024). The median PFS for all patients was 12.9 months (95%CI： 5.4~20.3 months). Patients receiving anti-angiogenic drugs had significantly longer PFS than those who did not (18.2 months vs 6.7 months， P=0.004). Univariate analysis showed that surgery， distant metastasis， and liver metastasis were significantly associated with PFS (all P<0.05). Multivariate Cox regression analysis indicated that treatment pattern was an independent prognostic factor for PFS (HR=5.228， 95%CI： 1.357~20.145). Subgroup analysis revealed no statistically significant difference in PFS among different anti-angiogenic drugs (anlotinib， apatinib， bevacizumab) or their timing of use (all P>0.05). Safety analysis showed generally manageable adverse events in both groups， the incidence of hypertension was higher in the treatment group (31.3% vs 0， P=0.033). No treatment-related death was observed in either group. ［Conclusion］ Anti-angiogenic drug treatment is associated with longer PFS in patients with advanced DSRCT and has a manageable safety profile， suggesting potential clinical value in the treatment of advanced DSRCT. |
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