Efficacy of sirolimus for epileptic seizures in childhood associated with focal cortical dysplasia type II.

Abstract

OBJECTIVE:

The efficacy of the mechanistic target of rapamycin inhibitor, sirolimus, was recently reported for patients more than 6 years of age by Kato et al. We evaluated the efficacy and safety of sirolimus in a 2-year-old patient with recurrent focal seizures with impaired consciousness after focal cortical dysplasia (FCD) type IIa resection.

METHODS:

The patient was a 2-year-old girl who had recurrent seizures after undergoing FCD resection at 4 months of age. The initial dose of sirolimus was 0.5 mg/day and was gradually increased using the trough blood concentration before oral administration as an index, and evaluation was performed at 92 weeks.

RESULTS:

The trough blood level of sirolimus was increased to 6.1 ng/mL and maintenance therapy was started at 40 weeks. Focal seizures with impairment of consciousness with tonic extension of the limbs decreased. No critically serious adverse events occurred.

CONCLUSION:

Sirolimus was effective against epileptic seizures from FCD type II even for a child under 5 years of age. There were no critically serious adverse events and administration could be continued.

• Authors: Hideaki Shiraishi, Tsuyoshi Teramoto, Saki Yokoshiki, Jun Tohyama, Yuki Ueda, Kiyoshi Egawa, Norihiro Sato, Atsushi Manabe, Mitsuhiro Kato
• Journal: Brain & development (Brain Dev) Vol. 45 Issue 6 Pg. 343-347 (Jun 2023) ISSN: 1872-7131 [Electronic] Netherlands
• PMID: 36870920 (Publication Type: Case Reports)
• Copyright: Copyright © 2023 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.

Chemical References

• Sirolimus

Topics

• Child
• Female
• Humans
• Child, Preschool
• Sirolimus (therapeutic use, pharmacology)
• Focal Cortical Dysplasia
• Seizures (drug therapy, etiology, surgery)
• Epilepsy (drug therapy, etiology, surgery)
• Epilepsy, Generalized
• Malformations of Cortical Development (complications, drug therapy)
• Magnetic Resonance Imaging