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The effect of everolimus on renal angiomyolipoma in pediatric patients with tuberous sclerosis being treated for subependymal giant cell astrocytoma

Pediatric Nephrology Nov 30, 2017

Bissler JJ, et al. - This post hoc analysis was carried out to determine the impact of everolimus on renal angiomyolipoma in pediatric patients with tuberous sclerosis being treated for subependymal giant cell astrocytomas (SEGAs) during the phase 3, randomized, double-blind, placebo-controlled EXamining everolimus In a Study of Tuberous sclerosis complex (EXIST)-1 trial. The findings of this analysis demonstrated the long-term efficacy and safety of everolimus as a therapeutic option for renal angiomyolipoma in this patient population, however, everolimus is currently not indicated for this use.
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