A Case Report on the Effect of Everolimus in Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex

Huma M Paika, Yull E. Arriaga, Anthony Setiawan


Tuberous Sclerosis (TS) is a well known disorder which manifests with benign tumors in various organ systems, including angiomyolipomas (AML) of the kidneys. The mTOR inhibitors have emerged as a potential systemic treatment strategy to halt the growth of renal angiomyolipomas and to prevent life threatening complications such as bleeding. This report shows that everolimus treatment may have an impact on size and density of renal AML lesions which may lead to prevention or delay the development of renal cell carcinoma as well as prevent bleeding from the renal AML lesions. A 20-year-old Hispanic female and a 24-year-old Asian female both with sporadic TS were administered daily oral everolimus therapy. Both showed stability in their renal AML lesions and notable improvement of their extra-renal TS manifestations. Treatment with daily everolimus may lead to reduction in size and/or decreases in density or renal AML lesions. This may have an impact on the risk of developing renal cell carcinoma as well as preventing bleeding from renal AML lesions.

J Med Cases. 2014;5(3):129-136
doi: http://dx.doi.org/10.14740/jmc1665w


mTOR inhibitor; Tuberous sclerosis; Angiomyolipoma; Everolimus

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