학술논문
Phase II study of alisertib as a single agent for treating recurrent or progressive atypical teratoid/rhabdoid tumor.
Document Type
article
Author
Upadhyaya, Santhosh; Campagne, Olivia; Billups, Catherine; Orr, Brent; Onar-Thomas, Arzu; Tatevossian, Ruth; Mostafavi, Roya; Myers, Jason; Vinitsky, Anna; Moreira, Daniel; Lindsay, Holly; Kilburn, Lindsay; Baxter, Patricia; Smith, Amy; Crawford, John; Partap, Sonia; Bendel, Anne; Aguilera, Dolly; Nichols, Kim; Rampersaud, Evadnie; Ellison, David; Klimo, Paul; Patay, Zoltan; Robinson, Giles; Broniscer, Alberto; Stewart, Clinton; Wetmore, Cynthia; Gajjar, Amar
Source
Neuro-Oncology. 25(2)
Subject
Language
Abstract
BACKGROUND: Recurrent atypical teratoid/rhabdoid tumor (AT/RT) is, most often, a fatal pediatric malignancy with limited curative options. METHODS: We conducted a phase II study of Aurora kinase A inhibitor alisertib in patients aged 12 months. PFS did not differ by AT/RT molecular groups. Neutropenia was the most common adverse effect (n = 23/30, 77%). The 22 patients who received liquid formulation had a higher mean maximum concentration (Cmax) of 10.1 ± 3.0 µM and faster time to Cmax (Tmax = 1.2 ± 0.7 h) than those who received tablets (Cmax = 5.7 ± 2.4 µM, Tmax = 3.4 ± 1.4 h). CONCLUSIONS: Although the study did not meet predetermined efficacy end point, single-agent alisertib was well tolerated by children with recurrent AT/RT, and SD or PR was observed in approximately a third of the patients.