Effectiveness of mTOR inhibitors in tuberous sclerosis complex-associated seizures
| Author, year | Medication | Condition | Patients, n | Results |
|---|---|---|---|---|
| Krueger et al, 2010 | Everolimus | TSC-associated SEGA | 16 | Improved seizure control over 34.2 months. Patients with no seizures since last visit increased from 38.5% to 65.2%. |
| Krueger et al, 2013 | Everolimus | TSC-associated refractory epilepsy | 20 | Well-tolerated with only mild and moderate adverse effects. Duration–dependent mechanism. |
| Cardamone et al, 2014 | Everolimus and Sirolimus | TSC-associated refractory epilepsy | 7 | One, four and two patients had >90%, 50%–90% and <50% reduction in seizure frequency over 18 months (median), respectively. |
| Overwater et al, 2016 | Sirolimus (adjunctive) | TSC-associated refractory epilepsy | 23 (children) | Despite seizure frequency reduction, significant benefits could not be proven. Lacked precision to exclude sirolimus benefits. |
| French et al, 2016 | Everolimus (adjunctive, low/high exposure) | TSC-associated refractory epilepsy | 366 (aged 2–65 years old) | Greater response rate, median reduction in seizure frequency and number of seizure-free days. Duration response and dose response. |
SEGA = subependymal giant-cell astrocytoma; TSC = tuberous sclerosis complex.