Abstract: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 reduce the surgical morbidity. Methods: One TSC-AML patient was treated with Everolimus and subsequently underwent resection. Toxicities, surgical complications, and tumor responses were monitored. Results: The patient received 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 period of 12 months. Conclusions: The administration of preoperative Everolimus therapy can impact the size and density of the primary tumor and appears safe and feasible. More TSC-AML patients can receive nephron-sparing partial nephrectomy.
[1]Curatolo P, Bombardieri R, Jozwiak S. Tuberous sclerosis. Lancet, 2008,372(9639):657-668. [2]Baskin HJ Jr. The pathogenesis and imaging of the tuberous sclerosis complex. Pediatr Radiol, 2008,38(9):936-952. [3]郭刚,蔡伟,高江平,等.新辅助分子靶向治疗在肾细胞癌患者治疗中的临床应用研究.微创泌尿外科杂志,2013,2(5):347-350. [4]Halpenny D, Snow A, McNeill G, et al. The radiological diagnosis and treatment of renal angiomyolipoma-current status. Clin Radiol, 2010,65(2):99-108. [5]Nelson CP, Sanda MG. Contemporary diagnosis and management of renal angiomyolipoma. J Urol, 2002,168(4 Pt 1):1315-1325. [6]Soulen MC, Faykus MH Jr, Shlansky-Goldberg RD, et al. Elective embolization for prevention of hemorrhage from renal angiomyolipomas. J Vasc Interv Radiol, 1994,5(4):587-591. [7]Yamakado K, Tanaka N, Nakagawa T, et al. Renal angiomyolipoma: relationships between tumor size, aneurysm formation, and rupture. Radiology, 2002,225(1):78-82. [8]Siroky BJ, Yin H, Bissler JJ. Clinical and molecular insights into tuberous sclerosis complex renal disease. Pediatr Nephrol, 2011,26(6):839-852. [9]Huang J, Manning BD. The TSC1-TSC2 complex: a molecular switchboard controlling cell growth. Biochem J, 2008,412(2):179-190. [10]Bissler JJ, Kingswood JC, Radzikowska E, et al. Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet, 2013,381(9869):817-824 [11]Staehler M, Sauter M, Helck A, et al. Nephron-sparing resection of angiomyolipoma after sirolimus pretreatment in patients with tuberous sclerosis. Int Urol Nephrol, 2012,44(6):1657-1661. [12]Krueger DA, Northrup H; International Tuberous Sclerosis Complex Consensus Group. Tuberous sclerosis complex surveillance and management: recommendations of the 2012 International Tuberous Sclerosis Complex Consensus Conference. Pediatr Neurol, 2013,49(4):255-226.