SAN FRANCISCO—Everolimus is the first potential pharmacologic treatment option for patients with angiomyolipoma (AML), a benign tumor that usually occurs in the kidney, researchers reported at the 2012 Genitourinary Cancers Symposium.

An international team conducted a prospective, double-blind study of 118 patients with AML randomized to receive everolimus 10 mg daily (79 patients) or placebo (39 patients). The study population included patients who had tuberous sclerosis complex or lymphangioleiomyomatosis. The primary efficacy endpoint was the proportion of patients with an AML response, defined as 50% or greater reduction in the sum of volumes of all target AML relative to baseline.

The mean duration of treatment was 38.1 weeks for the everolimus group and 34 weeks fo the placebo group. Results showed that 41.8% of everolimus-treated patients had an AML response compared with none of the placebo recipients. Additionally, the mean time to AML progression was 11.4 months for placebo and was not reached in the everolimus group. Everolimus-treated subjects also had significantly higher best overall skin lesion response rate (complete or partial response) than placebo (26% vs. 0%).

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The drug was associated with an acceptable safety profile consistent with previous reports in tuberous sclerosis complex. Most adverse effects were grade 1 or 2.

Angiomyolipomas rarely cause symptoms, but they can bleed or grow large enough to cause pain or renal failure.