Acute management of symptomatic subependymal giant cell astrocytoma with everolimus

Publication date: Available online 18 April 2017
Source:Pediatric Neurology
Author(s): Monica S. Arroyo, Darcy A. Krueger, Eileen Broomall, Charles B. Stevenson, David Neal Franz
Subependymal giant cell astrocytomas (SEGA) are slow-growing tumors which can cause obstructive hydrocephalus in patients with tuberous sclerosis complex (TSC). These tumors require routine surveillance with magnetic resonance imaging. Current consensus guidelines recommend treatment of asymptomatic SEGAs with mTOR inhibitor therapy, as these medications have demonstrated efficacy and safety in multiple prospective clinical trials. For symptomatic SEGAs, standard therapy typically involves surgical resection of the tumor to relieve mass effect and resolve hydrocephalus. However, resection can be associated with significant perioperative morbidity and complications. There are anecdotal reports of using mTOR inhibitors to reduce tumor size in preparation for surgery, but prospective studies comparing sole mTOR inhibitor therapy with surgical management have not been completed. Here we present a case of a 7-year old patient with a large, symptomatic SEGA which was successfully treated with everolimus, resulting in rapid reduction in tumor size, symptomatic improvement, and decrease in cerebrospinal fluid (CSF) protein, allowing for successful ventriculoperitoneal shunt placement without the need for surgical resection.



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