1. Phase II study of alisertib as a single agent for treating recurrent or progressive atypical teratoid/rhabdoid tumor.
- Author
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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, and Gajjar, Amar
- Subjects
Aurora kinase A ,alisertib ,alisertib pharmacokinetics ,atypical teratoid/rhabdoid tumor ,Child ,Humans ,Antineoplastic Agents ,Rhabdoid Tumor ,Azepines ,Pyrimidines ,Central Nervous System Neoplasms ,Aurora Kinase A ,Protein Kinase Inhibitors - 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.
- Published
- 2023