Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia: To Whom, When, and How

Allogeneic hematopoietic stem cell transplant (HSCT) is an established treatment modality with curative potential for acute myeloid leukemia (AML). There has been a significant rise in the number of HSCT procedures performed over the past decade due in part to improved supportive care and innovative techniques such as reduced-intensity conditioning. Expanding alternative donor options such as umbilical cord blood and haploidentical HSCT, taken together with improved outcomes of matched unrelated donors, has resulted in a suitable donor for most patients with an HSCT indication. Recent advances in molecular diagnostics that incorporate genetic mutational analysis into existing cytogenetic-based models should improve selection of patients at high risk of relapse most likely to benefit from HSCT. Improvements in minimal residual disease monitoring hold promise for adding prognostic information, and informing the clinician of impending relapse. The choice of the conditioning regimen involves weighing a patient's unique toxicity and relapse risks. Despite improvements, relapse remains the primary source of treatment failure after HSCT for treatment of AML. The introduction of novel therapies into the clinic, together with improved patient selection, offers hope for decreasing relapse and improving outcomes for AML patients.