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Blood, 1 July 2006, Vol. 108, No. 1, pp. 45-51.
Prepublished online as a Blood First Edition Paper on January 10, 2006; DOI 10.1182/blood-2005-08-3294.


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Submitted August 15, 2005
Accepted December 19, 2005

Clofarabine and cytarabine combination as induction therapy for acute myeloid leukemia (AML) in patients ≥ 50 years

Stefan Faderl*, Srdan Verstovsek, Jorge Cortes, Farhad Ravandi, Miloslav Beran, Guillermo Garcia-Manero, Alessandra Ferrajoli, Zeev Estrov, Susan O'Brien, Charles Koller, Francis J Giles, William Wierda, Monica Kwari, and Hagop M Kantarjian

Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

* Corresponding author; email: sfaderl{at}mdanderson.org.

Outcome of patients with acute myeloid leukemia (AML) over age 60 remains unsatisfactory with low remission rates and poor overall survival. We have previously established the activity of clofarabine plus cytarabine in AML relapse. We now conducted a phase 2 study of clofarabine plus cytarabine in patients ≥ 50 years with previously untreated AML. Clofarabine was given at 40mg/m2 as a 1-hour intravenous infusion for 5 days (days 2 to 6) followed 4 hours later by cytarabine at 1g/m2/day as a 2-hour intravenous infusion for 5 days (days 1 to 5). Of 60 patients, 29 (48%) had secondary AML, 30 (50%) had abnormal karyotypes (monosomy 5 and/or 7 in 15 [25%]), and 11 (21%) showed Flt3 abnormalities. The overall response rate (OR) was 60% (52% CR, 8% CRp). Four patients (7%) died during induction. Adverse events were mainly ≤ grade 2 and included diarrhea, nausea/vomiting, mucositis, skin reactions, liver test abnormalities, and infusion-related facial flushing and headaches. Myelosuppression was common. Clofarabine plus cytarabine has activity in adult AML achieving a good CR rate. However, survival does not appear to be improved compared to other regimens. Modifications of this combination in AML therapy of older patients warrant further evaluation.


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