Building: Bourbon Cataratas Convention Centre, Foz do Iguaçu
Room: Iguazu II
Date: 2014-05-08 02:15 PM – 02:30 PM
Last modified: 2014-02-09
Abstract
Introduction
Clinical manifestations of TSC increase over time and may begin in utero. Everolimus, an mTOR inhibitor, was superior to placebo for reducing SEGA volume in the EXIST-1 trial (NCT00789828; median age, 9.5 years; response rate: everolimus, 35% vs placebo, 0.0%; P˂0.0001; original cut-off, 02-March-2011). The safety of everolimus among patients ˂3 years old was evaluated in the open-label, long-term extension phase of EXIST-1 (28.3 months’ follow-up [range, 1.9-38.8 months]; cut-off, 11-January-2013).
Methods
Patients with ≥1 SEGA (≥1 cm in longest diameter) received 4.5 mg/m2/day oral everolimus (target trough, 5-15 ng/mL). Adverse events (AEs) were graded according to NCI-Common Terminology Criteria for Adverse Events version 3.0. We report safety data for patients ˂3 years old at the start of everolimus treatment (n=18; 12 males, 6 females).
Results
As of 11-January-2013, median duration of everolimus exposure in patients ˂3 years old at the start of treatment was 31.1 months (range, 11.5-39.0 months). The incidence of serious AEs was 50.0% (n=9); incidence of grade 3-4 AEs was 77.8% (n=14). Incidence of AEs requiring dose interruption and/or reduction was 94.4% (n=17). Most common AEs (occurring in ˃40%) were stomatitis (66.7%), cough (44.4%), pharyngitis (44.4%), and pyrexia (44.4%).
Conclusion
No new safety concerns were identified among patients ˂3 years old treated with everolimus. The small sample size in this subpopulation limits interpretation of the results.