Treatment of blast phase chronic myeloid leukaemia: A rare and challenging entity

Abstract

SummaryDespite the success of BCR‐ABL‐specific tyrosine kinase inhibitors (TKIs) such as imatinib in chronic phase (CP) chronic myeloid leukaemia (CML), patients with blast phase (BP)‐CML continue to have a dismal outcome with median survival of less than one year from diagnosis. Thus BP‐CML remains a critical unmet clinical need in the management of CML. Our understanding of the biology of BP‐CML continues to grow; genomic instability leads to acquisition of mutations which drive leukaemic progenitor cells to develop self‐renewal properties, resulting in differentiation block and a poor‐prognosis acute leukaemia which may be myeloid, lymphoid or bi‐phenotypic. Similar advances in therapy are urgently needed to improve patient outcomes; however, this is challenging given the rarity and heterogeneity of BP‐CML, leading to difficulty in designing and recruiting to prospective clinical trials. This review will explore the treatment of BP‐CML, evaluating the data for TKI therapy alone, combinations with intensive chemotherapy, the role of allogeneic haemopoietic stem cell transplantation, the use of novel agents and clinical trials, as well as discussing the most appropriate methods for diagnosing BP and assessing response to therapy, and factors predicting outcome.