Background
renal angiomyolipoma, SEGA, epilepsy, rAML, TSC
RENAL ANGIOMYOLIPOMA, SUBEPENDYMAL GIANT CELL ASTROCYTOMA, EPILEPSY, TUBEROUS SCLEROSIS COMPLEX, ADVERSE EFFECTS
The randomised double-blinded placebo-controlled EXIST-1–3 studies have showed everolimus effective with adverse effects reported as acceptable in treatment of symptoms in patients with tuberous sclerosis complex (TSC), although evidence of outcomes in clinical practice remains limited. This study aimed to investigate, in clinical practice, the effectiveness and safety of everolimus for epilepsy, renal angiomyolipoma (rAML), and subependymal giant cell astrocytoma (SEGA) in patients with TSC.