Vascular endothelial growth factor-targeted therapy for the treatment of adult metastatic Xp11.2 translocation renal cell carcinoma.
Vascular endothelial growth factor-targeted therapy for the treatment of adult metastatic Xp11.2 translocation renal cell carcinoma.
复制标题
DOI：
10.1002/cncr.25512
复制
发表时间：
2010-11-15
期刊：
影响因子：
6.2
通讯作者：
Tannir NM
中科院分区：
文献类型：
--
作者：
Choueiri TK;Lim ZD;Hirsch MS;Tamboli P;Jonasch E;McDermott DF;Dal Cin P;Corn P;Vaishampayan U;Heng DY;Tannir NM
成人“易位”肾细胞癌（RCC），承载TFE 3基因融合在Xp11.2，是最近公认的独特的实体，其预后和治疗仍然知之甚少。我们研究了血管内皮生长因子（VEGF）靶向治疗在这种不同亚型的肾细胞癌中的作用。我们进行了一项回顾性研究，以描述转移性Xp11.2 RCC成人患者的临床特征和结局，这些患者具有强TFE-3核免疫染色，并接受抗VEGF治疗。通过RECIST评价抗VEGF治疗的肿瘤反应。Kaplan-Meier方法用于估计无进展生存期（PFS）和总生存期（OS）分布。确定了15例患者，其中10例、3例和2例分别接受舒尼替尼、索拉非尼和抗VEGF单克隆抗体治疗。中位随访时间为19.1个月，患者的中位年龄为41岁，女性：男性比例为4：1。初始组织学描述包括透明细胞（n=8）、乳头状（n=1）或混合透明细胞/乳头状RCC（n=6）。5例患者既往接受过全身治疗。5例患者进行了FISH分析，均显示涉及染色体Xp11.2的易位。当接受VEGF靶向治疗时，3例患者部分缓解，7例患者病情稳定，5例患者病情进展。整个队列的中位PFS和OS分别为7.1个月和14.3个月。成年期易位相关转移性肾细胞癌是一种侵袭性疾病，影响年轻患者人群，女性占优势。VEGF靶向药物在这个小型回顾性系列中表现出一定的疗效。
Adult “translocation” renal cell carcinoma (RCC), bearing TFE3 gene fusions at Xp11.2, is a recently recognized unique entity for which prognosis and therapy remain poorly understood. We investigated the effect of vascular-endothelial growth factor (VEGF)-targeted therapy in this distinct subtype of RCC. We conducted a retrospective review to describe the clinical characteristics and outcome of adult patients with metastatic Xp11.2 RCC, who had strong TFE-3 nuclear immunostaining, and received anti-VEGF therapy. Tumor response to anti-VEGF therapy was evaluated by RECIST. Kaplan-Meier methods were used to estimate progression-free survival (PFS) and overall survival (OS) distributions. Fifteen patients were identified of which 10, 3, and 2 received sunitinib, sorafenib and monoclonal anti-VEGF antibodies, respectively. The median follow-up was 19.1 months, the median age of the patients was 41 years, and the female:male ratio was 4:1. Initial histologic description included clear cell (n=8), papillary (n=1) or mixed clear cell/papillary RCC (n=6). Five patients had prior systemic therapy. Five patients had FISH analysis and all demonstrated a translocation involving chromosome Xp11.2. When treated with VEGF-targeted therapy, 3 patients had a partial response, 7 patients had stable disease and 5 patients had progressive disease. The median PFS and OS of the entire cohort were 7.1 months and 14.3 months respectively. Adult-onset translocation-associated metastatic RCC is an aggressive disease that affects a younger population of patients with a female predominance. VEGF-targeted agents demonstrated some efficacy in this small retrospective series.