BRAIN and NERVE Shinkei Kenkyu no Shinpo Volume 71, Issue 6 (June 2019)

The Efficacy of Everolimus for Refractory Seizures in Childhood Onset Epilepsy with Tuberous Sclerosis Complex Tohru Okanishi 1 , Ayataka Fujimoto 2 , Mitsuyo Nishimura 3 , Shinpei Baba 1 , Shinji Itamura 1 , Kazuo Okanari 4 , Hideo Enoki 1 1Department of Child Neurology, Comprehensive Epilepsy Center, Seirei Hamamatsu General Hospital 2Department of Epilepsy and Surgery, Comprehensive Epilepsy Center, Seirei Hamamatsu General Hospital 3Division of Laboratory, Seirei Hamamatsu General Hospital 4Department of Pediatrics, Oita University Keyword: 結節性硬化症 , てんかん , エベロリムス , mTOR阻害薬 , tuberous sclerosis complex , epilepsy , everolimus , mTOR inhibitor pp.611-616
Published Date 2019/6/1
  • Abstract
  • Look Inside
  • Reference


Everolimus is a mammalian target of rapamycin (mTOR) inhibitor that has cytoreductive effects on subependymal giant cell astrocytoma and renal angiomyolipoma in tuberous sclerosis complex (TSC). Recent studies have also shown its efficacy against refractory seizures in TSC. We investigated the efficacy of everolimus in nine patients with TSC, who were admitted to the TSC clinic in Seirei Hamamatsu General Hospital and who suffered from refractory seizures. At the start of treatment, patients ranged from 1 month to 23 years of age, and were refractory to a mean of 5.4 antiepileptic agents. Main seizures were focal in six patients and generalized in three patients. After 0.5 to 4.0 years (mean=2.4 years), three patients (33%) were seizure-free and two patients (22%) experienced >90% reduction in seizures. Everolimus may therefore be effective in the treatment of refractory seizures in TSC.

(Received February 20, 2019; Accepted April 2, 2019; Published June 1, 2019)

Copyright © 2019, Igaku-Shoin Ltd. All rights reserved.


BRAIN and NERVE-神経研究の進歩
71巻6号 (2019年6月)
電子版ISSN 1344-8129 印刷版ISSN 1881-6096 医学書院