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:https://doi.org/10.1016/j.seizure.2020.03.018
Highlights
•
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
Abstract
Purpose
This study aimed to analyze the therapeutic effect of
sirolimus on seizures in pediatric patients with tuberous sclerosis.
Methods
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.
Results
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.
Conclusions
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.
Courtesy of: https://www.mdlinx.com/journal-summaries/seizure-disorders-seizures-tuberous-sclerosis-complex/2020/04/28/7670287?spec=neurology
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