TSC – Everolimus for Seizures — SCE Neurology MCQ
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Correct answer: A — Everolimus
Everolimus (an mTOR inhibitor) has been approved for the treatment of refractory seizures associated with TSC (EXIST-3 trial). TSC is caused by mutations in TSC1 or TSC2, leading to overactivation of the mTOR pathway. Everolimus directly targets this pathological mechanism. It can reduce seizure frequency in patients who are not surgical candidates or as an adjunct to surgery. A: Temozolomide is for gliomas. C: Sirolimus (rapamycin) is a related mTOR inhibitor used for some TSC manifestations but everolimus has the specific seizure indication. D: Bevacizumab is for GBM. E: Cyclophosphamide is for autoimmune/oncological conditions.
Reference: NICE TA687 Everolimus for TSC-related Seizures; EXIST-3 Trial (2016)