Acute Management of Symptomatic Subependymal Giant Cell Astrocytoma With Everolimus

Pediatric Neurology
Monica S ArroyoD N Franz

Abstract

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 an mechanistic target of rapamycin (mTOR) inhibitor because 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 seven-year-old boy with a large, symptomatic SEGA which was treated acutely with everolimus. Everolimus treatment resulted in rapid reduction in tumor size, symptomatic improvement, and decrease in cerebrospinal fluid protein. Everolimus can effectively reduce tumor size, decrease cerebrospinal fluid prot...Continue Reading

Citations

Oct 12, 2018·American Journal of Medical Genetics. Part C, Seminars in Medical Genetics·David Neal Franz, Darcy Andrew Krueger
Sep 25, 2019·Cancers·Periklis KatopodisEmmanouil Karteris
Apr 28, 2020·Expert Opinion on Pharmacotherapy·Daniel Ebrahimi-Fakhari, David Neal Franz
Mar 6, 2021·Neuro-oncology Practice·James S RyooAnkit I Mehta
Aug 17, 2021·Pediatric Neurology·Hope NorthrupUNKNOWN International Tuberous Sclerosis Complex Consensus Group

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