Allogeneic Stem Cell Transplantation for Myelodysplastic Syndrome

Although it is only used to treat a minority of patients with myelodysplastic syndromes, stem cell transplantation (SCT) is the only proven curative treatment for this condition. Because MDS occurs in a population of older adults with significant comorbidities, reduced-intensity conditioning (RIC) regimens have been particularly important in extending safe SCT to the large MDS population over the age of 60 years. Extension of the unrelated donor pool together with the introduction of umbilical cord blood transplants in adults has extended the number of patients with suitable donors. Nevertheless overall mortality from SCT is greater than 50% because of relapse and non-relapse mortality (NRM). New developments to improve outcome include the tailoring of the transplant approach to the individual based on age and comorbidity, and the use of pretransplant chemotherapy to reduce disease bulk prior to transplant, as well as the introduction of post-transplant immunotherapy (pre-emptive donor lymphocyte infusions) and chemotherapy to prevent relapse. Further improvements in transplant outcome await better ways to reconstitute immunity and amplify the graft-versus-leukemia (GVL) effect without causing graft-versus-host disease (GVHD), as well as further extension of the donor pool and exploration of risk-adapted regimens for the population of MDS in their seventh to eighth decade.