Phase 2 clinical and pharmacologic study of clofarabine in patients with refractory or relapsed acute leukemia

Hagop Kantarjian, Varsha Gandhi, Jorge Cortes, Srdan Verstovsek, Min Du, Guillermo Garcia-Manero, Francis Giles, Stefan Faderl, Susan O'Brien, Sima Jeha, Jan Davis, Zeev Shaked, Adam Craig, Michael Keating, William Plunkett, Emil J. Freireich

Research output: Contribution to journalArticlepeer-review

152 Scopus citations


In a phase 2 study, 62 patients with relapsed and refractory acute myeloid leukemia (AML; n = 31), myelodysplastic syndrome (MDS; n = 8), chronic myeloid leukemia in blastic phase (CMLBP; n = 11), and acute lymphocytic leukemia (ALL; n = 12) received 40 mg/m2 clofarabine intravenously over 1 hour daily for 5 days, every 3 to 6 weeks. Twenty patients (32%) achieved complete response (CR), 1 had a partial response (PR), and 9 (15%) achieved CR but without platelet recovery (CRp), for an overall response rate of 48%. In AML, responses were noted in 2 (18%) of 11 patients in first salvage with short first CR (≤ 12 months), in 7 (87%) of 8 patients with longer first CR, and in 8 (67%) of 12 patients in second or subsequent salvage. Responses were observed in 4 of 8 patients with high-risk MDS (50%), in 7 (64%) of 11 with CML-BP, and in 2 (17%) of 12 with ALL. Severe reversible liver dysfunction was noted in 15% to 25%. After the first clofarabine infusion, responders accumulated more clofarabine triphosphate in blasts compared with nonresponders (median 18 vs 10 μM; P = .03). This increased only in responders (median, 1.8-fold; P = .008) after the second clofarabine infusion. In summary, clofarabine is active in acute leukemias and MDS; cellular pharmacokinetics may have prognostic significance.

Original languageEnglish (US)
Pages (from-to)2379-2386
Number of pages8
Issue number7
StatePublished - Oct 1 2003
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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