Genotype and subependymal lesion burden influence volumetric response to everolimus in tuberous sclerosis complex-associated subependymal giant cell astrocytoma: A 10-year real-world study.

Abstract excerpt

Everolimus, a mechanistic target of rapamycin (mTOR) inhibitor, effectively reduces subependymal giant cell astrocytoma (SEGA) volume in patients with tuberous sclerosis complex (TSC); however, biological determinants of therapeutic sensitivity remain poorly defined. In this 10-year real-world cohort, we retrospectively evaluated 43 patients (median age 12 years; 46.5% female) with TSC-associated SEGA treated...

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