PubMed · 814264
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H Aziz. 1975. Epilepsy--I.. https://pubmed.ncbi.nlm.nih.gov/814264/
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BACKGROUND: Everolimus, a mechanistic target of rapamycin (mTOR) inhibitor, is increasingly used in tuberous sclerosis complex (TSC)-associated epilepsy; however, long-term real-world outcomes and factors associated with favorable response remain unclear. This study aimed to evaluate the long-term seizure outcomes of adjunctive everolimus and explore clinical factors associated with treatment response. METHODS: We retrospectively recruited 21 patients with active TSC-associated epilepsy receiving adjunctive everolimus and assessed seizure outcomes during follow-up. Clinical characteristics were compared between responders and non-responders at 1 year after treatment initiation. RESULTS: Over a median treatment duration of 72 months, responder rates ranged from 53.8% to 64.7%, and seizure-free rates ranged from 33.3% to 41.2%. Responders had fewer involved organ systems at baseline (median 3 vs. 4, p = 0.020) and lower anti-seizure medication burden (median 2 vs. 4, p = 0.045). Younger age at treatment initiation showed a trend toward improved response. CONCLUSION: Adjunctive everolimus was associated with sustained long-term seizure reduction in this real-world cohort. In exploratory analyses, fewer involved organ systems and fewer baseline ASMs were associated with favorable treatment response. These findings require validation in larger prospective cohorts.
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Epilepsy is one the commonest neurological diseases. In 25% of the cases, particularly in patients with temporal foci, the seizures prove to be resistant to medicinal treatment. Several investigations have stressed that these patients have pronounced psychosocial problems which may be more important to the patient than the actual seizures. Taking into consideration the WHO definition of health as complete physical, mental and social well-being, patients with epilepsy have problems in all of these dimensions. The major problems are social isolation and employment. Surgical treatment of epilepsy has a pronounced effect on the seizures in 90% of the operations in the best series but it is still uncertain whether reduction in the number of seizures also implies better quality of life. The present data suggest improvement in the psychosocial conditions of the patients who functioned best preoperatively and who became seizure-free after operation. The importance of further longitudinal studies emphasizing the psychosocial aspects is stressed.