Chronic myeloid leukemia: mechanisms of blastic transformation

J Clin Invest. 2010 Jul;120(7):2254-64. doi: 10.1172/JCI41246. Epub 2010 Jul 1.

Abstract

The BCR-ABL1 oncoprotein transforms pluripotent HSCs and initiates chronic myeloid leukemia (CML). Patients with early phase (also known as chronic phase [CP]) disease usually respond to treatment with ABL tyrosine kinase inhibitors (TKIs), although some patients who respond initially later become resistant. In most patients, TKIs reduce the leukemia cell load substantially, but the cells from which the leukemia cells are derived during CP (so-called leukemia stem cells [LSCs]) are intrinsically insensitive to TKIs and survive long term. LSCs or their progeny can acquire additional genetic and/or epigenetic changes that cause the leukemia to transform from CP to a more advanced phase, which has been subclassified as either accelerated phase or blastic phase disease. The latter responds poorly to treatment and is usually fatal. Here, we discuss what is known about the molecular mechanisms leading to blastic transformation of CML and propose some novel therapeutic approaches.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Leukemia / drug therapy*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy*
  • Lymphocyte Activation
  • Mice
  • Protein Kinase Inhibitors / therapeutic use*

Substances

  • Protein Kinase Inhibitors