Allogeneic stem cell transplantation for patients with myelodysplastic syndromes

Abstract

Myelodysplastic syndromes (MDS) are a heterogenous group of clonal hematopoietic stem cell neoplasms primarily affecting older persons, associated with dysplastic changes of bone marrow cells, peripheral cytopenias, and various risk of leukemic transformation. Although treatment with several drugs has shown improved disease control, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment for MDS. The number of patients receiving a transplant, as well as survival, have increased past years because of the use of reduce-intensity conditioning regimens (RIC) as well as the use of haploidentical donors for transplantation. With treatment-related mortality as main limitation, pre-transplant evaluation is essential to assess risks for this older group of patients. In a recent randomized study, allo-HSCT with RIC for patients >50 years old with higher-risk MDS demonstrated superiority in survival compared with hypomethylating agents. Genetic mutations have been shown to significantly impact treatment outcomes including after transplant. Recently, a transplant-specific risk score (which includes age, donor type, performance status, cytogenetic category, recipient's cytomegalovirus status, percentage of blasts, and platelet count) has shown superiority in transplantation outcome prediction, compared with previous scoring systems. Survival remains low for most patients with TP53 mutations and novel treatment strategies are needed, such as administration of natural killer cells post-transplant, as there is no clear evidence that maintenance therapy after transplantation can improve outcomes.