The Role of Hematopoietic Stem Cell Transplantation in CML

Hematopoietic stem cell transplantation (HSCT) remains a very important tool in the therapeutic armamentarium of chronic myeloid leukemia. Although the numbers of transplant performed today represent approximately 15% of those undertaken before the tyrosine kinase inhibitors became widely used in clinical practice, this significant minority can benefit very considerably from HSCT. Whereas no physician nor patient would welcome unnecessary risk in the chronic phase, there are some patients in whom HSCT provides a greater probability of long-term survival than the continuation of ineffective drugs. The goal is to recognize these patients and to refer them for transplant before disease progression. For the unfortunate patient who presents in or progresses to a blast crisis, HSCT is the only therapy to offer the possibility of cure. Risk factors for outcome after HSCT are well known and can be estimated on an individual basis, providing the transplant equivalent of “precision medicine” in a disease that has benefited so markedly from targeted therapy. Close interaction between CML physicians and transplant centers facilitates this personalized risk-adapted approach.