Sunday, May 3, 2020

Sirolimus treats epilepsy in pediatric patients with tuberous sclerosis

Wen He, Jian Chen, Yang-Yang Wang, Xiao-Mei Luo, Xiao-Qiao Chen, Li-Ping Zou.  Sirolimus Improves Seizure Control in Pediatric Patients with Tuberous Sclerosis: a Prospective Cohort Study.  Seizure. Published:April 26, 2020DOI:

Sirolimus has a significant effect on seizures associated with tuberous sclerosis
Early use of sirolimus to control seizures can bring long-term benefits
The overall tolerability of sirolimus is acceptable


This study aimed to analyze the therapeutic effect of sirolimus on seizures in pediatric patients with tuberous sclerosis.

We first compared the efficacy of controlling seizures in all patients after they had taken sirolimus for one year, and then we performed a subgroup analysis based on whether the administered antiepileptic drugs were changed to determine whether the efficacy was associated with changes of antiepileptic drugs.

A total of 91 eligible children were enrolled. The response rate was 78.0% (71/91), and 47.2% (43/91) of all patients were became seizure-free. The improvement in seizure control before and after treatment with sirolimus was significant ( p < 0.001). In the AEDs unaltered group, 34 were responders (34/45, 75.6%, 95% CI 17.4–88.3), of which 24 were seizure-free (24/34, 70.6%). In the AEDs-altered group, 37 were responders (37/46, 80.4%, 95% CI 56.7–88.1), of which 19 were seizure-free (19/37, 51.4%). There was no significant difference between the two groups for reductions in rate of seizure frequency ( p = 0.308). In the patients with refractory epilepsy, treatment with sirolimus was also effective ( p = 0.01). Logistic regression analysis showed that age was an important factor affecting outcome of epilepsy ( p = 0.003, 95% CI 2.05–38.31). No Grade 3 or 4 adverse events were noted during the follow-up.

Sirolimus has a significant effect on seizures associated with tuberous sclerosis complex (TSC), with no or only moderate adverse events after long-term administration. Sirolimus could be used as the first-line medication for pediatric patients with TSC-associated epilepsy.

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