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

• Type of Material: Article
• 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
• Language: English
• Date: 2014-10-01
• Publisher: Lippincott, Williams & Wilkins
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2013 by Lippincott Williams & Wilkins.
• Final Published Version (URL): http://dx.doi.org/10.1097/MPH.0000000000000005
• Title of Journal or Parent Work: Journal of Pediatric Hematology/Oncology
• ISSN: 1077-4114
• Volume: 36
• Issue: 7
• Start Page: E448
• End Page: E451
• Grant/Funding Information: R.C.C.: Received funding from NIH R01CA172392, a Scholar Award (St. Baldrick’s Foundation), Springboard Grant (Alex’s Lemonade Stand Foundation), and Research Grants (Childhood Brain Tumor Foundation, CURE Childhood Cancer Foundation, and the Children’s Center for Neuroscience Pilot Grant).
• Keywords: Life Sciences & Biomedicine; everolimus; PATIENT; Science & Technology; SIROLIMUS; Pediatrics; Oncology; TUMORS; TUBEROUS SCLEROSIS; MODEL; tuberous sclerosis; SEGA; spinal cord; Hematology
• Research Categories: Biology, Neuroscience; Health Sciences, Pathology; Health Sciences, Radiology

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.