Sirolimus for epileptic seizures associated with focal cortical dysplasia type II.

ABSTRACT:

Objective

To determine whether sirolimus, a mechanistic target of rapamycin (mTOR) inhibitor, reduces epileptic seizures associated with focal cortical dysplasia (FCD) type II.

Methods

Sixteen patients (aged 6-57 years) with FCD type II received sirolimus at an initial dose of 1 or 2 mg/day based on body weight (FCDS-01). In 15 patients, the dose was adjusted to achieve target trough ranges of 5-15 ng/mL, followed by a 12-week maintenance therapy period. The primary endpoint was a lower focal seizure frequency during the maintenance therapy period. Further, we also conducted a prospective cohort study (RES-FCD) in which 60 patients with FCD type II were included as an external control group.

Results

The focal seizure frequency reduced by 25% in all patients during the

SUBMITTER: Kato M 

PROVIDER: S-EPMC8862414 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature