Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex
Mary Kay Koenig1, Ian J Butler, Hope Northrup
1Department of Pediatrics, Division of Child and Adolescent Neurology, The University of Texas Health Science Center, Houston, Texas, USA. mary.k.koenig@uth.tmc.edu
Abstract:
The authors present a 21-year-old woman who has been receiving rapamycin for 5 months for bilateral subependymal giant cell astrocytomas. The patient was started at a dose of 0.2 mg/kg/day. Levels were maintained between 11 and 13 ng/mL. Magnetic resonance imaging of the brain 2(1/2) months after initiating rapamycin demonstrated a decrease in size of both astrocytomas (11 to 7.5 mm on the right and 8 to 5 mm on the left). Further studies are needed with prolonged observation to confirm these findings, determine the length of necessary treatment, and evaluate recurrence risk after discontinuation of rapamycin.

