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 2½ 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.
Kenerson H., Dundon TA, Yeung RSEffects of rapamycin in the Eker rat model of tuberous sclerosis complex. Pediatr Res. 2005;57:67-75.
2.
Rauktys A., Lee N., Lee L., Dabora SLTopical rapamycin inhibits tuberous sclerosis tumor growth in a nude mouse model. BMC Dermatol. 2008;8:1-9.
3.
Bissler JJ, McCormack FX, Young LR, et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 ;358:140-151.
4.
Franz DN, Leonard J., Tudor C., et al. Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol. 2006;59:490-498.
