Alisertib Is Active as Single Agent in Recurrent Atypical Teratoid Rhabdoid Tumors in Four Children

The study from 2015 investigated the efficacy of alisertib, an Aurora Kinase A inhibitor, in treating four pediatric patients with recurrent atypical teratoid rhabdoid tumors (ATRT). The children, with a median age of 2.5 years, were administered alisertib at a dose of 80 mg/m² orally once daily for 7 days in a 21-day cycle. After three cycles, all patients showed either disease stabilization or regression, with two patients maintaining stable disease for 1 and 2 years. The treatment resulted in 261 adverse events, including 42 grade 4 events, with common toxicities being somnolence, alopecia, neutropenia, decreased WBCs, thrombocytopenia, and anemia. Despite these toxicities, the study concluded that alisertib is a tolerable and effective treatment for ATRT in children, warranting its inclusion in future clinical trials.