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- W865158342 abstract "We evaluated prospectively the effect of Everolimus on epilepsy in children and adolescents with TSC treated with Everolimus for SEGA at our center. Methods This is a retrospective analysis of prospectively collected data: included were patients ≤19 years with epilepsy, treated with Votubia, who had regular prospective follow-up every 3 months at the study center (clinical inspection and EEG, seizure diaries, blood sampling). Everolimus is orally administered once per day, starting with 4.5 mg/m 2 and titration to achieve blood trough concentrations of 5–15 ng/ml. Response was defined as ≥50% reduction in seizure count. Results We present pilot data of 12 patients (4 female, 8 male), with genetically confirmed diagnosis of TSC (3 TSC1, 9 TSC2 mutations). The median age at initiation of Votubia was 10 years (y) (range: 2–19 y). The median duration of epilepsy prior to treatment was 9.5 y (range: 1.75–16.75 y). The median number of AEDs prior to treatment was 1 (range: 1–3). The median seizure frequency prior to treatment was 30 seizures/month (range: 2–1470 seizures/month). 8/12 (66%) patients were responders. The median follow up after initiation of Everolimus was 13.5 months (range: 2–31 months). The median reduction in seizure frequency was 93% (range: 0–100%). Four patients became seizure free. The median number of AED after treatment was 1 (range: 1–2). The effect on seizure frequency was accompanied by significant developmental progress in all seizure free children. 4 patients were non-responders, one of them was incompliant. Side effects were observed in 7 patients (58%). The most common side effects were aphtae (7/7); 1 patient was hospitalized because of angina herpetica and pneumonia. In no case the Everolimus treatment had to be interrupted or withdrawn. Conclusions Everolimus seems to be effective and safe in treating TSC associated epilepsy in pediatric patients (even in cases prior refractory to AEDs)." @default.
- W865158342 created "2016-06-24" @default.
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- W865158342 date "2015-05-01" @default.
- W865158342 modified "2023-09-27" @default.
- W865158342 title "OP53 – 2462: The role of mTOR inhibitors in TSC associated epilepsy" @default.
- W865158342 doi "https://doi.org/10.1016/s1090-3798(15)30054-4" @default.
- W865158342 hasPublicationYear "2015" @default.
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