依维莫司在结节性硬化合并肾血管平滑肌脂肪瘤术前新辅助治疗中的应用

Presurgical targeted therapy with Everolimus for angiomyolipoma associated with tuberous sclerosis complex

目的:探讨依维莫司在结节性硬化合并肾血管平滑肌脂肪瘤术前新辅助治疗中的疗效与安全性。方法:对1例结节性硬化合并双肾血管平滑肌脂肪瘤患者进行包括高选择性肾血管栓塞、肾部分切除术及依维莫司的综合治疗,对临床资料和随访情况进行回顾性分析并文献复习。结果:该患者接受了高选择性肾血管栓塞术加肾部分切除术治疗右肾血管平滑肌脂肪瘤,术后应用依维莫司治疗2个月,左肾肿瘤最大直径缩小60%(5.1cm),停药3个月后接受左肾部分切除术,痊愈出院,随访12个月,无肿瘤复发及进展。结论:结节性硬化症合并双肾血管平滑肌脂肪瘤的患者治疗必须考虑肾功能的保留问题,对于一些无法行保留肾单位手术的患者,可以考虑在术前行依维莫司新辅助治疗,使患者获得手术的机会。

Objective:To evaluate the feasibility and safety of a presurgical treatment approach with Everolimus for angiomyolipoma (AML)associated with tuberous sclerosis complex (TSC)and explore its potential ability to re-duce the surgical morbidity.Methods:One TSC-AML patient was treated with Everolimus and subsequently under-went resection.Toxicities,surgical complications,and tumor responses were monitored.Results:The patient re-ceived super-selective renal arterial embolization combined with nephron-sparing resection for right side renal tumor. Then,she received Everolimus (10 mg Qd)for two months.The change in primary renal tumors was a 60% (5.1 cm)decrease after two months.The patient received drug discontinuance for 3 months before operation.She received nephron sparing surgery for her left side renal tumor.There was no recurrence or progression during a follow-up pe-riod of 12 months.Conclusions:The administration of preoperative Everolimus therapy can impact the size and densi-ty of the primary tumor and appears safe and feasible.More TSC-AML patients can receive nephron-sparing partial nephrectomy.