Long-term treatment of epilepsy with everolimus in tuberous sclerosis.

ABSTRACT:

Objective

To evaluate the long-term benefit and safety of everolimus for the treatment of medically refractory epilepsy in patients with tuberous sclerosis complex (TSC).

Methods

Everolimus was titrated over 4 weeks and continued an additional 8 weeks in a prospective, open-label, phase I/II clinical trial design. Participants demonstrating initial benefit continued treatment until study completion (48 months). The primary endpoint was percentage of patients with a ≥50% reduction in seizure frequency compared to baseline. Secondary endpoints assessed absolute seizure frequency, adverse events (AEs), behavior, and quality of life.

Results

Of the 20 participants who completed the initial study phase, 18 continued extended treatment. Fourteen of 18 (78%) participants com

SUBMITTER: Krueger DA 

PROVIDER: S-EPMC5177677 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature