The Contemporary Role of Hematopoietic Stem Cell Transplantation in the Management of Chronic Myeloid Leukemia: Is It the Same in All Settings?

Simple Summary This review explores the use of hematopoietic stem cell transplantation (HSCT) as a treatment option for chronic myeloid leukemia (CML) patients. While CML treatment has greatly improved with tyrosine kinase inhibitors (TKIs), some patients do not respond well or reach advanced disease stages. The goal now is to achieve treatment-free remission (TFR). Although discontinuing TKIs shows promise, relapse risk is high. This review discusses recent advances in HSCT and its role in CML treatment, suggesting it should be considered early in disease management to enhance the chances of achieving TFR alongside TKIs.Abstract Hematopoietic stem cell transplantation (HSCT) for chronic myeloid leukemia (CML) patients has transitioned from the standard of care to a treatment option limited to those with unsatisfactory tyrosine kinase inhibitor (TKI) responses and advanced disease stages. In recent years, the threshold for undergoing HSCT has increased. Most CML patients now have life expectancies comparable to the general population, and therefore, the goal of therapy is shifting toward achieving treatment-free remission (TFR). While TKI discontinuation trials in CML show potential for achieving TFR, relapse risk is high, affirming allogeneic HSCT as the sole curative treatment. HSCT should be incorporated into treatment algorithms from the time of diagnosis and, in some patients, evaluated as soon as possible. In this review, we will look at some of the recent advances in HSCT, as well as its indication in the era of aiming for TFR in the presence of TKIs in CML.