中文摘要：

目的 观察晚期肾透明细胞癌（clear cell renal cell carcinoma,ccRCC）中血管内皮生长因子（vascular endothelial growth factor,VEGF）和血小板衍化生长因子（platelet-derived growth factor,PDGF）相关因子的表达,并分析其表达量与舒尼替尼远期疗效的相关性。方法 选取2010年1月—12月复旦大学附属中山医院收治的转移性晚期肾癌患者20例。所有患者均予患肾根治性切除术,病理证实均为ccRCC。所有患者术后均予舒尼替尼标准给药方案治疗,50 mg每天1次口服,服药4周、停药2周为1个疗程,每6周随访1次,随访至2015年12月底。免疫组化方法检测入组患者病理标本肿瘤组织中VEGF-A、VEGF-C、VEGFR-2、VEGFR-3、PDGF和PDGFR-α、β的表达水平,计算肿瘤微血管密度（microvessel density,MVD）,并与靶向治疗远期疗效进行相关性分析。结果 截至研究终点日期,所有患者均未出现因不能控制的严重不良反应而停药的情况,存活4人,死亡16人,死亡患者均因肿瘤进展导致。Spearman分析显示VEGFR-2高表达与较长的无进展生存时间（progression free survival,PFS）及总生存时间（overall survival,OS）存在显著相关,单因素Cox生存回归分析中VEGFR-2强阳性与肿瘤进展风险降低（OR=0.162,95%CI：0.031-0.839,P=0.030）及死亡风险降低（OR=0.204,95%CI：0.041-1.015,P=0.052）具有较为显著的相关性。结论 接受舒尼替尼治疗的晚期ccRCC患者中,VEGFR-2高表达与较长的PFS及OS存在显著相关,VEGFR-2强阳性与肿瘤进展风险降低及死亡风险降低显著相关,提示VEGFR-2有可能成为晚期肾癌靶向治疗的远期疗效预测因子。

英文摘要：

Objective To explore the expressions of vascular endothelial growth factor （VEGF） and platelet-derived growth factor（PDGF）,and their relationship with prospective efficacy of tyrosine kinase inhibitor sunitinib in metastatic clear cell renal cell carcinoma （ccRCC）. Methods Twenty patients with histologically proven metastatic ccRCC were selected between Jan., 2010 and Dec., 2010.All patients underwent radical nephrectomy and then were followed up to Dec.,2015.All patients received a standard regimen of sunitinib （a dose of 50 mg given orally once daily for 4 weeks on and 2 weeks off,6 weeks per cycle）.Tissue microarrays from primary tumor specimens of all patients were immunohistochemically stained for selected markers, including PDGF,PDGFR-α,β,VEGF-A,VEGF-C and VEGFR-2,3.And the results of immunohistochemistry （IHC） and microvessel density （MVD） were compared with patients＇ survival. Results To the date of statistical analysis,16 patients died of tumour progression.High expression of VEGFR-2 was significantly correlated with the longer progression free survival （PFS） and overall survival （OS） in Spearman correlation analysis.Cox regression analysis revealed that a strong positive of VEGFR-2 was significantly associated with both lower risk of tumor progressing（OR=0.162,95%CI：0.031-0.839,P=0.030） and lower mortality risk （OR=0.204,95%CI：0.041-1.015,P=0.052）. Conclusions For ccRCC patients who treated with sunitinib,high expression of VEGFR-2 was significantly correlated with the longer PFS and OS,while a strong positive of VEGFR-2 was significantly associated with both lower risk of tumor progressing and lower mortality risk.VEGFR-2 could be a predictive factor for making a clinical decision in RCC targeted therapy.