Stem Cell Transplantation and Cellular Immunotherapies for Pediatric AML

During the past three decades, allogeneic hematopoietic stem cell transplantation (HSCT) has significantly contributed to the improvement of survival in patients with high-risk acute myeloid leukemia (AML). Although transplant-related mortality occurs in ~5% of patients, HSCT is still considered the only available curative option for most patients with high-risk AML. Many factors such as the stem cell donor source, conditioning regimen, and time of transplantation contribute to the success of HSCT. Clinical experience gained across international transplant trials indicates the best conditions, which will be reviewed in this chapter. However, regardless of the improvements achieved in treatment, many children with AML have primary chemotherapy-refractory disease or experience a subsequent relapse. Alternative therapies, including immunotherapies, are thus needed to overcome chemoresistance that leads to relapse and/or poor clinical outcomes. Significant progress in the immunotherapy field has led to clinical investigation of new antibody-based and cellular-based therapies in the field of adult AML. Some of these modalities are now under pediatric-specific evaluation with several additional therapies under study in the near future. This chapter further provides an overview of available and soon-to-be-explored immunotherapies in clinical trials for pediatric patients with relapsed/refractory AML.