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Blood. 2015 May 7;125(19):2923-32. doi: 10.1182/blood-2014-10-608117. Epub 2015 Mar 24.

Vosaroxin and vosaroxin plus low-dose Ara-C (LDAC) vs low-dose Ara-C alone in older patients with acute myeloid leukemia.

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Department of Haematology, Christie Hospital, Manchester, United Kingdom;
Department of Haematology, Nottingham University Hospital National Health Service Trust, Nottingham, United Kingdom;
Department of Haematology, Cardiff University School of Medicine, Cardiff, United Kingdom;
Department of Haematology, Queen's Hospital, Romford, United Kingdom;
Department of Haematology, Imperial College, Northwick Park Hospital Campus, London, United Kingdom;
Department of Haematology, Belfast City Hospital, Belfast, United Kingdom;
Department of Haematology, Rigshospitalet, Copenhagen, Denmark;
Department of Haematology, Queen Alexandra Hospital, Portsmouth, United Kingdom;
Department of Haematology, Royal Liverpool University Hospital, Liverpool, United Kingdom; and.
Department of Haematology, Heartlands Hospital, Birmingham, United Kingdom.


The development of new treatments for older patients with acute myeloid leukemia is an active area, but has met with limited success. Vosaroxin, a quinolone-derived intercalating agent has several properties that could prove beneficial. Initial clinical studies showed it to be well-tolerated in older patients with relapsed/refractory disease. In vitro data suggested synergy with cytarabine (Ara-C). To evaluate vosaroxin, we performed 2 randomized comparisons within the "Pick a Winner" program. A total of 104 patients were randomized to vosaroxin vs low-dose Ara-C (LDAC) and 104 to vosaroxin + LDAC vs LDAC. When comparing vosaroxin with LDAC, neither response rate (complete recovery [CR]/complete recovery with incomplete count recovery [CRi], 26% vs 30%; odds ratio [OR], 1.16 (0.49-2.72); P = .7) nor 12-month survival (12% vs 31%; hazard ratio [HR], 1.94 [1.26-3.00]; P = .003) showed benefit for vosaroxin. Likewise, in the vosaroxin + LDAC vs LDAC comparison, neither response rate (CR/CRi, 38% vs 34%; OR, 0.83 [0.37-1.84]; P = .6) nor survival (33% vs 37%; HR, 1.30 [0.81-2.07]; P = .3) was improved. A major reason for this lack of benefit was excess early mortality in the vosaroxin + LDAC arm, most obviously in the second month following randomization. At its first interim analysis, the Data Monitoring and Ethics Committee recommended closure of the vosaroxin-containing trial arms because a clinically relevant benefit was unlikely.

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