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Objectives To evaluate the effects and utility of intermittent everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex. Methods We investigated a total of 26 patients with tuberous sclerosis complex who had angiomyolipoma ≥4 cm in diameter. For each patient, we analyzed the reduction in the size of the angiomyolipoma, the change in size after everolimus withdrawal, the size reduction rate on everolimus readministration and adverse events caused by everolimus. The volume of angiomyolipoma was measured using abdominal computed tomography or magnetic resonance imaging. Adverse events were evaluated according to CTCAE v4.0-JCOG. Results The average size reduction rate of angiomyolipoma in the initial treatment with everolimus was 67%. Eight patients (31%) did not have enlarged angiomyolipoma after everolimus withdrawal. The other 18 patients (69%) restarted everolimus treatment because of enlargement of the angiomyolipoma. The average size reduction rate of angiomyolipoma in the everolimus retreatment group was 61%, which was equivalent to the rate for the initial treatment. There were fewer adverse events during everolimus retreatment than in the initial treatment. Conclusions This is the first report regarding intermittent everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex. This treatment is effective for tumor control and adverse event management. This beneficial treatment option for patients can minimize the drug dosage and the occurrence of adverse events. [ABSTRACT FROM AUTHOR]

Objectives To evaluate the efficacy and toxicity profiles of everolimus in Japanese patients with renal angiomyolipoma associated with tuberous sclerosis complex. Methods Patients with a 4-cm or larger angiomyolipoma meeting the diagnostic criteria of tuberous sclerosis complex were selected as participants of our investigation. In each case, we assessed tuberous sclerosis complex-associated symptoms, the treatment effect and adverse events. The treatment effect was evaluated by measuring the tumor volume reduction rate using abdominal computed tomography or magneitc resonance imaging. Adverse events were investigated using CTCAE v4.0- JCOG. The dose of everolimus was set at 10 mg once a day for adults. For childhood angiomyolipoma, everolimus administration was initiated at a dose of 5 mg once a day. Blood everolimus level was measured, and the dose was adjusted to maintain this within a range of 5-15 ng/ mL. Results The angiomyolipoma volume decreased in 46 of 47 cases, and the mean reduction rate for all cases was 60% in 12 months. The angiomyolipoma volume markedly decreased in the first 3 months, and the size leveled off after 6 months. The main adverse events related to everolimus treatment were stomatitis (91%) and irregular menstruation (65%). Grade 3 or severer adverse events were noted in three cases (6%). All patients developed some adverse events in the first 6 months. The incidence markedly decreased to 40-50% after 13 months. Conclusion The tumor volume-reducing effect of everolimus in a Japanese population was equivalent to or higher than that in Western populations. A wide variety of everolimus treatment-related adverse events can be observed, but most cases are very mild. Special attention should be given to stomatitis, irregular menstruation and interstitial lung disease as adverse events. [ABSTRACT FROM AUTHOR]