Novel agents targeting leukemia cells and immune microenvironment for prevention and treatment of relapse of acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation

Shi, Wei; Jin, Weiwei; Xia, Linghui; Yu, Hu

Relapse remains the worst life-threatening complications after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with acute myeloid leukemia (AML), whose prognosis has been historically dismal. Given the rapid development of genomics and immunotherapies, the interference strategies for AML recurrence have been changing these years. More and more novel targeting agents that have received the U.S. Food and Drug Administration (FDA) approval for de novo AML treatment have been administrated in the salvage or maintenance therapy of post-HSCT relapse. Targeted strategies that regulate the immune microenvironment of and optimize the graft versus leukemia (GVL) effect of immune cells are gradually improved. Such agents not only have been proven to achieve clinical benefits from a single drug, but if combined with classic therapies, can significantly improve the poor prognosis of AML patients who relapse after allo-HSCT. This review will focus on currently available and promising upcoming agents and also discuss the challenges and limitations of targeted therapies in the allogeneic hematopoietic stem cell transplantation community.