Adjunctive everolimus therapy for treatment-resistant focal-onset seizures associated with tuberous sclerosis (EXIST-3): a phase 3, randomised, double-blind, placebo-controlled study

French J. A., Lawson J. A., Yapici Z., Ikeda H., Polster T., Nobbout R., ...More

LANCET, vol.388, no.10056, pp.2153-2163, 2016 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 388 Issue: 10056
  • Publication Date: 2016
  • Doi Number: 10.1016/s0140-6736(16)31419-2
  • Journal Name: LANCET
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.2153-2163
  • Istanbul University Affiliated: Yes

Abstract

Background Everolimus, a mammalian target of rapamycin (mTOR) inhibitor, has been used for various benign tumours associated with tuberous sclerosis complex. We assessed the efficacy and safety of two trough exposure concentrations of everolimus, 3-7 ng/mL (low exposure) and 9-15 ng/mL (high exposure), compared with placebo as adjunctive therapy for treatment-resistant focal-onset seizures in tuberous sclerosis complex.