Early everolimus boosts tumour control in TSC-related angiomyolipoma

In patients with tuberous sclerosis complex (TSC), early everolimus intervention for neuronal lesions leads to better tumour control of renal angiomyolipoma (AML) and lower side effects, a recent study has found.

The study included 20 TSC patients who were treated with everolimus for their disease-related epilepsy and subependymal giant cell astrocytoma. All patients also had small AMLs <3 cm. Everolimus was initially dosed at 3 mg/m² once per day and eventually adjusted to maintain a blood trough level of 5–15 ng/mL. Treatment was halted when an adverse event grade ≥3 arose.

Patients were treated with everolimus for a median of 24 months, during which time 30 percent (n=6) achieved complete remission. All cases of complete remission occurred within 6–9 months after everolimus initiation, stayed on treatment, and had not relapsed by study end.

Of note, the total target AML volume had been significantly reduced within 3 months of everolimus treatment (p=0.005), and all patients achieved a reduction to <50 percent. The mean maximum reduction ratio in total target AML volume was 79 percent.

In terms of safety, the researchers reported that all adverse events were grade 1 or 2 in severity. The most common side effects were stomatitis (65 percent) and irregular menstruation (18 percent).

“Early intervention with everolimus may cure small AMLs associated with TSC. This treatment option can minimize drug dosage and the occurrence of adverse events,” the researchers said.