Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma.

ABSTRACT:

Objective

To report long-term efficacy and safety data for everolimus for the treatment of subependymal giant cell astrocytoma (SEGA) in patients with tuberous sclerosis complex (TSC).

Methods

This was an open-label extension phase of a prospective, phase 1-2 trial (NCT00411619) in patients ≥3 years of age with SEGA associated with TSC. Patients received oral everolimus starting at 3 mg/m2 per day and subsequently titrated, subject to tolerability, to attain whole blood trough concentrations of 5-15 ng/mL. Change in SEGA volume, seizures, and safety assessments were the main outcome measures.

Results

Of 28 patients enrolled, 25 were still under treatment at the time of analysis. Median dose was 5.3 mg/m2/day and median treatment duration was 34.2 months (range 4.7-47.

SUBMITTER: Krueger DA 

PROVIDER: S-EPMC3589289 | biostudies-literature | 2013 Feb

REPOSITORIES: biostudies-literature