Molecular biology and treatment of CML.

Abstract Since the development of imatinib mesylate, an ABL tyrosine kinase inhibitor (TKI), the treatment of chronic myeloid leukemia (CML) has made remarkable progress. Second and third generation TKIs have also become available for clinical use. Considering the improved treatment outcomes, chronic phase CML has become manageable, with a low mortality rate. Furthermore, clinical trials such as STIM and DADI have shown that TKI can be discontinued safely in certain CML patients. However, some CML patients, especially those in accelerated phase (AP) or blast phase (BP), require more aggressive forms of treatment such as allogenic hematopoietic stem cell transplantation. Next-generation sequencing technology and other novel technologies allow us to investigate the cause of CML and the molecular biology of progression to AP/BP in more detail. Based on these data, an increasing number of novel therapeutic agents for CML are being developed, which will improve the prognosis of CML.