Everolimus Alleviates Obstructive Hydrocephalus Due to Subependymal Giant Cell Astrocytomas

Publication date: Available online 3 January 2017
Source:Pediatric Neurology
Author(s): Romina Moavero, Andrea Carai, Angela Mastronuzzi, Sara Marciano, Federica Graziola, Federico Vigevano, Paolo Curatolo
Background and purposeSubependymal giant cell astrocytomas (SEGA) are low-grade tumors affecting up to 20% of patients with tuberous sclerosis complex (TSC). Early neurosurgical resection has been the only standard treatment until few years ago when the better understanding of the molecular pathogenesis of TSC led to the use of mTOR inhibitors. Surgical resection of SEGA is still considered as the first line treatment in individuals with symptomatic hydrocephalus and intratumoral hemorrhage. We report a clinical series of 4 patients presenting symptomatic or asymptomatic hydrocephalus who were successfully treated with everolimus.Main findingsAll patients presented a clinically significant response to everolimus and an early shrinkage with improvement of ventricular dilatation. Everolimus appeared to be well tolerated in all subjects.ConclusionOur clinical series demonstrate a possible expanding indication for mTOR inhibition in TSC, which can be considered in patients with asymptomatic hydrocephalus or even when the symptoms already appeared. It offers a significant therapeutic alternative to individuals that once would have undergone immediate surgery. Everolimus can allow to postpone neurosurgical resection, making it elective with an overall lower risk.



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