Targeted therapies in hematology and their impact on patient care: chronic and acute myeloid leukemia

Semin Hematol. 2013 Oct;50(4):271-83. doi: 10.1053/j.seminhematol.2013.09.006. Epub 2013 Oct 3.


Advances in the genetic and molecular characterizations of leukemias have enhanced our capabilities to develop targeted therapies. The most dramatic example of targeted therapy in cancer to date is the use of targeted BCR-ABL protein tyrosine kinase inhibitors (TKI), which has revolutionized the treatment of chronic myeloid leukemia (CML). Inhibition of the signaling activity of this kinase has proved to be a highly successful treatment target, transforming the prognosis of patients with CML. In contrast, acute myeloid leukemia (AML) is an extremely heterogeneous disease with outcomes that vary widely according to subtype of the disease. Targeted therapy with monoclonal antibodies and small molecule kinase inhibitors are promising strategies to help improve the cure rates in AML. In this review, we will highlight the results of recent clinical trials in which outcomes of CML and AML have been influenced significantly. Also, novel approaches to sequencing and combining available therapies will be covered.

Publication types

  • Review

MeSH terms

  • Antibodies, Monoclonal / therapeutic use
  • Clinical Trials as Topic
  • Humans
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Molecular Targeted Therapy*
  • Patient Care
  • Protein Kinase Inhibitors / therapeutic use


  • Antibodies, Monoclonal
  • Protein Kinase Inhibitors