Treatment options for chronic myeloid leukemia

The bcr-abl tyrosine kinase inhibitors (TKIs) are the cornerstone treatment for chronic myeloid leukemia (CML). However, there are many topics related to therapy that remain debated.

The aim of this paper is to give the reader a comprehensive review of how to treat CML at diagnosis, how to monitor the disease and a brief read of special populations and case scenarios. It describes the first-line (imatinib) and second-line (nilotinib and dasatinib) TKIs currently used for the treatment of CML, including landmark studies proving their efficacy, side effect profile, dosage and use in special populations. It also reviews the current guidelines regarding treatment and monitoring of the disease while on TKIs, along with an overview of treatment in advanced stages, the role of allogeneic stem cell transplantation and investigational drugs.

Although imatinib represented a mayor therapeutic advancement over conventional chemotherapy, second-generation TKIs offer higher rates of optimal response and should be used as the frontline therapy. Patients with the T315I mutation carry a worse prognosis and should be offered allogeneic stem cell transplantation. The treatment in advanced stages of CML remains suboptimal and bench, translational and clinical research is encouraged.