Are we making progress in GVHD prophylaxis and treatment?

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HCT) is an effective immunotherapy for human cancer. More than 20 000 allo-HCTs are performed each year worldwide, primarily for the treatment of hematologic malignancies. Several technical innovations implemented in allo-HCT over past 2 decades have reduced NRM by 50% and improved overall survival. The allo-HCT practice has changed with the introduction of peripheral blood, cord blood, and haploidentical transplantations and reduced-intensity conditioning, and the patient population is also different regarding age and diagnosis. However, both acute and chronic GVHD remain serious barriers to successful allo-HCT and it is not clear that a major improvement has occurred in our ability to prevent or treat GVHD. Nevertheless, there is an increasing knowledge of the biology and clinical manifestations and the field is getting better organized. These advances will almost certainly lead to major progress in the near future. As the long list of new potential targets and respective drugs are developed, systems need to be developed for rapid testing of them in clinical practice. The current reality is that no single agent has yet to be approved by the US Food and Drug Administration for GVHD prevention or therapy. Although a primary goal of these efforts is to develop better therapies for GVHD, the ultimate goal is to develop treatments that lead to effective prevention or preemption of life-threatening and disabling GVHD manifestations while harnessing the desirable graft-versus-tumor effects.