351. Sirolimus improves seizure control in pediatric patients with tuberous sclerosis: A prospective cohort study.
- Author
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He W, Chen J, Wang YY, Zhang MN, Qian-Lu, Wang QH, Luo XM, Chen XQ, and Zou LP
- Subjects
- Adolescent, Anticonvulsants administration & dosage, Child, Child, Preschool, Epilepsy etiology, Female, Humans, Infant, Male, Prospective Studies, Protein Kinase Inhibitors administration & dosage, Sirolimus administration & dosage, TOR Serine-Threonine Kinases antagonists & inhibitors, Tuberous Sclerosis complications, Anticonvulsants pharmacology, Epilepsy drug therapy, Outcome Assessment, Health Care, Protein Kinase Inhibitors pharmacology, Sirolimus pharmacology, Tuberous Sclerosis drug therapy
- 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., Competing Interests: Declaration of Competing Interest The authors declare that they have no competing interests., (Copyright © 2020. Published by Elsevier Ltd.)
- Published
- 2020
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