ICNC Abstracts, ICNC 2018

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Mammalian Target of Rapamycin (mTOR) Inhibitors as an Effective Therapy for Refractory Epilepsy associated with Tuberous Sclerosis Complex: A Systematic Review

Last modified: 2018-09-09


Purpose -   To systematically review the evidence on the efficacy and safety of mammalian target of rapamycin (mTOR) inhibitors in controlling intractable epilepsy associated with tuberous sclerosis complex.

Method: Literature reviewed using Medline searches, Cochrane Library and Biomed central with the keywords tuberous sclerosis, refractory epilepsy/SEGA and the text word mTOR. Bibliographies of papers located by searches and review articles were compiled. Papers published from various subcontinents worldwide, reporting use of rapamycin & its analogs (rapalogs) in tuberous sclerosis complex-related epilepsies were selected. Six studies met all criteria for inclusion. It included 3 prospective and 3 randomized controlled trials. Studies were reviewed for seizure frequency, seizure-free days and adverse effects. The outcome measures used were the percentage of patients with complete seizure-free, >50% reduction in seizures, 25-50% reduction in seizures and <25% or no effect.

Results: The estimated proportion for obtaining complete seizure free control by combined analysis (confidence profile method) was 17.84 % (95% confidence interval [CI]: 0.1184-0.2380) with 32.12% (95% CI: 0.2839-0.3585) reporting greater than 50% reduction in seizures. Common adverse events observed were upper respiratory tract infection, gastrointestinal problems, lipid abnormalities and some serious adverse events like otitis media, pneumonia were reported while on the treatment and during follow-up period. At our center out of two patients who received Everolimus, one became completely seizure free in 3 months duration.

Conclusion: There is strong evidence to support the safe and effective use of mTOR inhibitors in the treatment of tuberous sclerosis complex related epilepsy.


tuberous sclerosis, epilepsy,MTor

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