Clofarabine is approved by the Food and Drug Administration (FDA) to treat relapsed or refractory acute lymphoblastic leukemia (ALL) in pediatric patients who have already had at least two other types of treatment. Clofarabine is also being studied in the treatment of other types of cancer.
In a European multi-center study, clofarabine was administered first line, as a monotherapy, to 30 patients over 60 years of age with AML and 13 (43%) patients achieved a complete response (CR); 4 (13%) patients achieved a complete response without full platelet recovery (CRp); and 3 (10%) achieved a partial remission (PR), thus giving an overall response rate of 66%. This compared favorably with the current best standard of care, low-dose ara-C, which achieved a CR rate of 17% in the AML 14 Trial conducted by the UK National Cancer Research Institute. The duration of remission observed with clofarabine was also durable with the interim follow-up at a median of at least 6 months. An application has been submitted to the FDA for approval for adult AML. A phase II study concluded: “These interim data indicate that single agent CLO is active and well-tolerated in treatment-naïve, older AML patients with 1 adverse prognostic factor, especially unfavorable risk karyotype and/or AHD.”
There is another study going on: Clofarabine Combinations in Relapsed/Refractory AML, MDS and Myeloid Blast Phase CML, NCT00067028. There has been no phase III trial confriming that Clofarabine is superior to 7+3 in any setting.
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