Revisiting autologous transplantation in acute myeloid leukemia

Autologous hematopoietic cell transplantation (auto-HCT) has been evaluated as a consolidation treatment for acute myeloid leukemia (AML) in the 1980s and 1990s. These prospective studies from large trials compared auto-HCT with chemotherapy. A comparison was made also with allogeneic hematopoietic cell transplantation (allo-HCT). These studies reported a lower relapse rate with auto-HCT compared with chemotherapy, but without impact on the overall survival. A high transplant-related mortality in that era confounded the relevance of these data.

Several prospective studies and a plethora of retrospective registry data have confirmed the potent antileukemic therapy of auto-HCT compared with chemotherapy and, in some instances, have even challenged the presumed superiority of allo-HCT as the definitive therapy for certain patients with AML.

The aggregate of recent data, prospective and retrospective, strongly suggests an important role for auto-HCT, at least as the most potent nonimmunologic antileukemia therapy. The transplant-related mortality in 2017 is close to that expected from standard consolidation therapy leading to the conclusion that the role of auto-HCT needs to be rigorously revisited, preferably in prospective studies, to establish its precise role in the current era.