The current and future role of stem cells in myelodysplastic syndrome therapies

Allogeneic blood stem cell transplantation (aBSCT) still is the only curative therapy for patients with myelodysplastic syndromes. While it carries the hope for cure for some patients, it may result in severe toxicity and death from complications or recurrent disease in others. Recent developments have improved patient and donor selection as well as technical aspects of the transplant procedure and post-transplant care, including early detection and treatment of relapse. Areas covered: This review will discuss current stratification tools to identify suitable patients, donors and transplant techniques. In addition, it will cover the prognostic and predictive value of cytogenetics and somatic mutations and elucidate strategies for minimal residual disease detection as well as prophylactic and preemptive treatment of recurrent disease. Expert commentary: aBSCT will continue to be the most powerful curative treatment option for patients with MDS. Advances in the understanding of disease biology will allow to incorporate molecular alterations in current prognostic scores, tracking of specific mutations during therapy and the use of novel, targeted therapies to augment the graft versus leukemia effect.