PURPOSE OF REVIEW:
The only current treatment capable of curing patients with myelodysplastic syndromes (MDS) is allogeneic haematopoietic stem cell transplantation (HCT). However, many MDS patients are older, often with substantial comorbid conditions, and the disease is heterogeneous. As a consequence, results of HCT vary considerably, and the practices of HCT for MDS are evolving.
The newly published modified International Prognostic Scoring System (IPSS-R), developed for nontransplanted patients, also correlates with post-HCT outcome, with the patient's karyotype having the strongest impact. The presence of monosomal karyotype and various genetic and molecular markers have also been shown to have a prognostic value. The use of hypomethylating agents, before or after HCT, may reduce the post-HCT relapse risk or delay relapse. Low and reduced-intensity conditioning regimens have allowed to transplant growing numbers of older patients with MDS, and the development of novel regimens may lead to improved relapse-free survival even in patients with high-risk cytogenetics. The optimal stem cell source may differ for different patient populations and different disease risk categories.
Transplant results for MDS have improved in recent years. Some patients even in the eighth decade of life have been transplanted successfully. Ongoing studies are aimed at further reducing transplant-related toxicity, graft-versus-host disease and post-HCT relapse.