Approaches to the treatment of chronic myeloid leukemia

In the past two decades there have been a number of advances in the treatment of chronic myeloid leukemia (CML) in chronic phase. There has been very little progress in the management of advanced disease. Hydroxyurea and interferon-alpha have replaced busulfan as first-line therapy in chronic phase. Hydroxyurea provides excellent control of the disease for periods of months or years and has little toxicity. Interferon-alpha induces a reduction in the Philadelphia chromosome in a substantial minority of patients. Interferon-alpha may also prolong survival. Neither hydroxyurea nor interferon-alpha is able to cure the disease. Patients in chronic phase may be cured by allogeneic bone marrow transplantation (BMT). This procedure has a number of limitations, however, including limited availability, high cost, and substantial morbidity and mortality. Current challenges in BMT are to increase its applicability, to reduce graft-versus-host disease, and to augment graft-versus-leukemia effects. The role of autologous transplantation in CML remains speculative.