Stage I of a phase 2 study assessing the efficacy, safety, and tolerability of barasertib (AZD1152) versus low‐dose cytosine arabinoside in elderly patients with acute myeloid leukemia

BACKGROUND In this phase 2 study, the authors evaluated the efficacy, safety, and tolerability of the Aurora B kinase inhibitor barasertib compared with low‐dose cytosine arabinoside (LDAC) in patients aged ≥60 years with acute myeloid leukemia (AML). METHODS Patients were randomized 2:1 to receive either open‐label barasertib 1200 mg (as a 7‐day intravenous infusion) or LDAC 20 mg (subcutaneously twice daily for 10 days) in 28‐day cycles. The primary endpoint was the objective complete response rate (OCRR) (complete responses [CR] plus confirmed CRs with incomplete recovery of neutrophils or platelets [CRi] according to Cheson criteria [also requiring reconfirmation of CRi ≥21 days after the first appearance and associated with partial recovery of platelets and neutrophils]). Secondary endpoints included overall survival (OS) and safety. RESULTS In total, 74 patients (barasertib, n = 48; LDAC, n = 26) completed ≥1 cycle of treatment. A significant improvement in the OCRR was observed with barasertib (35.4% vs 11.5%; difference, 23.9%; 95% confidence interval, 2.7%‐39.9%; P