Response of Subependymal Giant Cell Astrocytoma With Spinal Cord Metastasis to Everolimus

• Authors: Dolly Aguilera, Emory University; Robert Castellino, Emory University; Claire Mazewski, Emory University; Matthew Schniederjan, Emory University; Laura Hayes, Children's Healthcare of Atlanta; William Boydston, Emory University; Robert C. Flamini, Children's Healthcare of Atlanta; Tobey MacDonald, Emory University
• Journal Title: Journal of Pediatric Hematology/Oncology
• Volume: Volume 36, Number 7
• Publisher: Lippincott, Williams & Wilkins | 2014-10-01, Pages E448-E451
• Type of Work: Article | Post-print: After Peer Review
• Publisher DOI: http://doi.org/10.1097/MPH.0000000000000005
• Subjects: Biology, Neuroscience; Health Sciences, Pathology; Health Sciences, Radiology
• Keywords: Science & Technology; Life Sciences & Biomedicine; Oncology; Hematology; Pediatrics; everolimus; SEGA; tuberous sclerosis; spinal cord; TUBEROUS SCLEROSIS; SIROLIMUS; PATIENT; TUMORS; MODEL

Abstract

Background: Brain subependymal giant cell astrocytomas (SEGAs) in patients with tuberous sclerosis have been reported to respond to everolimus.Methods: A 15-year-old male patient with intractable seizures and multiple SEGAs of the brain developed leptomeningeal enhancement and multiple metastatic, histologically confirmed SEGAs of the spinal cord. He received daily everolimus at a dose of 3 mg/m2 for 6 weeks, which was then increased to 6 mg/m2.Results: Magnetic resonance image of the brain and spine showed significant reduction in the size of SEGAs after 6 weeks of treatment. The patient has remained free of progression for 24 months. Additional benefits included: excellent seizure control, decrease in the size of cardiac rhabdomyomas, and improved quality of life.Conclusions: We describe a rare case of metastatic SEGA, which was successfully treated with everolimus.