Indications for Allogeneic Hematopoietic Cell Transplantation in Myelodysplastic Syndrome

Myelodysplastic syndromes (MDS) are heterogeneous diseases that principally affect older adults. Allogeneic hematopoietic cell transplantation (HCT) is the only potentially curative therapy; however, non-relapse mortality (NRM) accounts for as many as 40% of deaths after HCT and underscores the need for careful patient selection. We review the common indications and causes of failure after HCT in MDS.

Appropriate patient selection is necessary to optimize HCT outcomes and maximize the life-expectancies of MDS patients. The international prognostic scoring systems (IPSS) and revised IPSS (IPSS-R) are used to identify high-risk patients and guide decision making. Neither scoring system incorporates molecular mutations, which are now recognized as important predictors of disease biology and clinical outcomes. Patient and disease characteristics including age, comorbid conditions, iron overload, blast percentage, and other features may impact post-HCT outcomes. An accurate assessment of the disease risk and patient qualities that affect NRM is necessary to optimize post-HCT outcomes. In this review, we summarize the risk factors for, and common causes of NRM, as well as markers of poor-risk disease that should lead providers to consider allogeneic HCT in MDS patients.