Clofarabine: emerging role in leukemias

Expert Opin Investig Drugs. 2009 Oct;18(10):1559-64. doi: 10.1517/13543780903173222.


Clofarabine is a second-generation purine nucleoside analogue. It works mainly by inhibiting ribonucleotide reductase and incorporating into DNA. Clofarabine has shown efficacy in selected pediatric leukemias. It has also shown significant efficacy alone and in combination with other drugs in treating adult myeloid leukemias and high-risk myelodysplastic syndromes. Further, there is significant promise for clofarabine in the treatment of older patients with acute myeloid leukemia who are unlikely to benefit from standard induction chemotherapy due to unfavorable baseline prognostic factors. An oral formulation of clofarabine is also currently under development.

MeSH terms

  • Adenine Nucleotides / adverse effects
  • Adenine Nucleotides / pharmacology
  • Adenine Nucleotides / therapeutic use*
  • Adult
  • Aged
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Arabinonucleosides / adverse effects
  • Arabinonucleosides / pharmacology
  • Arabinonucleosides / therapeutic use*
  • Child
  • Clinical Trials as Topic
  • Clofarabine
  • Drug Evaluation, Preclinical
  • Humans
  • Leukemia / diagnosis
  • Leukemia / drug therapy*
  • Leukemia / physiopathology
  • Middle Aged
  • Prognosis


  • Adenine Nucleotides
  • Antineoplastic Agents
  • Arabinonucleosides
  • Clofarabine