Hematopoietic Cell Transplantation in Myelodysplastic Syndromes

This chapter provides case studies of patients with myelodysplastic syndromes (MDS) and their treatment, including hematopoietic stem cell transplantation (HSCT). Regarding the optimal intensity of the induction regimen, published data seem to favor a transplant performed with minimal burden of disease, especially in reduced-intensity conditioning (RIC) regimens that most heavily rely on the graft-versus-leukemia (GVL) effect. Lenalidomide has proved effective in MDS with del(5q); about two-thirds of patients become transfusion independent, with a median response duration of 2.2 years; it has also been shown to be effective in non-del(5q) MDS. In patients with progressive disease, as evidenced by cytogenetic progression and worsened cytopenias, it is reasonable to consider HSCT. If a matched related or matched unrelated donor is unavailable, transplant from a UCB or haploidentical donor is reasonable in very high-risk cases. Secondary MDS is an absolute indication for allogeneic HSCT, in light of the poor prognosis of this disease.