Everolimus Reduces the Size of Tuberous Sclerosis Complex-Related Huge Renal Angiomyolipomas Exceeding 20 cm in the Longest Diameter

We evaluated the efficacy of everolimus in 3 patients who had huge renal angiomyolipomas associated with tuberous sclerosis complex. Two patients with large lipid-rich angiomyolipomas had a history of renal transarterial embolization for renal bleeding, but the effect had only been temporary and the embolized kidneys had continued to enlarge. In case 1, case 2, and case 3, total renal volume was respectively 3,891, 4,035, and 1,179 cm 3 before administration of everolimus, decreasing to 3,016 (77%), 3,043 (75%), and 1,051 (89%) cm 3 after 1 year of everolimus therapy and to 2,832 (73%), 3,209 (80%), and 1,102 (93%) cm 3 after 3 years. New renal bleeding did not occur, but elevation of serum creatinine and urinary protein were noted in 2 patients. While previous reports have largely assessed the effect of everolimus for angiomyolipomas of < 10 cm in the longest diameter, our findings suggest that this drug might also be effective for huge lesions of > 20 cm in diameter. However, total renal volume still exceeds 2,000 cm 3 in 2 of our patients, suggesting limited size reduction of lipid-rich angiomyolipomas. In addition, occurrence of everolimus-related nephropathy needs to be monitored carefully.