Subependymal Giant Cell Astrocytoma Treatment Patterns among Patients with Tuberous Sclerosis Complex (P3.283)

Objective: To use the tuberous sclerosis complex (TSC) Natural History Database to describe subependymal giant cell astrocytoma (SEGA) treatment patterns among patients with TSC. Background: SEGAs are benign brain lesions that occur in 10[percnt] to 20[percnt] of TSC patients. Surgical resection has been the standard of care for SEGAs. In 2010, the first drug therapy, everolimus (an mTOR inhibitor), became available for the treatment of SEGA. Methods: This study analyzed TSC patients diagnosed with SEGA in the TSC Natural History Database, which contains demographics, clinical features, and treatment information for 1,328 TSC patients in the US. The analysis included all SEGA treatments following the diagnosis. We described the treatment sequences and assessed the median time between sequential treatments. Results: SEGA was diagnosed in 377 (28[percnt]) TSC patients. Among them, 51[percnt] were male; 76[percnt] were white, 14[percnt] were Hispanic, and 7[percnt] were African American. Median age at the time of diagnosis was 8.1, ranging from 0 to 48.3 years. A total of 181 patients (48[percnt]) had at least one treatment following diagnosis. Of these, 56[percnt] received a resection as the first treatment. In this group, 63[percnt] received no further treatment and 24[percnt] received at least one additional resection. Median time between the first resection and the second resection was 2.9 years. Of the 181 treated patients, seventy-two patients (40[percnt]) initiated an mTOR inhibitor as the first treatment; two (3[percnt]) had a subsequent resection. The first treatment of the remaining 8 treated patients (4[percnt]) was a shunt-related procedure. Conclusions: More than half of the patients with TSC diagnosed with SEGA received no SEGA treatment during the period of observation. Among treated patients, SEGA resection and mTOR inhibitor were the most common treatments. The overwhelming majority of patients who received an mTOR inhibitor did not receive any subsequent resection. Study supported by: Novartis Disclosure: Dr. Krueger has received research support from Novartis. Dr. Song has received personal compensation for activities with Novartis. Dr. Swallow has received personal compensation for activities with Novartis. Ms. King has received research support from Novartis. Dr. Peeples has received personal compensation for activities with Novartis. Dr. Signorovitch has received personal compensation from Novartis. Dr. Liu has nothing to disclose. Dr. Prestifilippo has nothing to disclose. Dr. Korf has received personal compensation in an editorial capacity for Annual Reviews of Genomics and Human Genetics. Dr. Sparagana has received personal compensation from Novartis and Lundbeck Pharmaceuticals. Dr. Wong has nothing to disclose. Dr. Kohrman has received personal compensation for activities with Novartis as a consultant.