Allogeneic hematopoietic cell transplantation for MDS: For whom, when and how?

Hematopoietic cell transplantation (HCT) is currently the only treatment with curative potential for patients with myelodysplastic syndrome (MDS). However, treatment-related mortality and relapse have remained major barriers to uniform success. Therefore, important questions remain to be answered, such as whom to transplant, when and how. With reduced intensity conditioning (RIC) regimens, patients in their 70s and patients with comorbid conditions have been transplanted successfully, although the relapse incidence with this approach tends to be increased in comparison to high intensity regimens. Success rates are higher in patients transplanted at an early stage of their disease. Encouraging is the fact, that results with unrelated donors who are HLA-matched by high resolution typing are comparable to those achieved with HLA genotypically identical siblings. The establishment of cord blood as a source of stem cells, and the recent success with HLA-haploidentical related donors will allow the offering of HCT to virtually all patients. Dependent upon disease stage and characteristics, some 25% to 75% of transplanted patients will be cured. While 20%–30% of patients experience chronic medical problems after HCT, 70% report a “good to excellent” quality of life. New studies must focus on further reducing GVHD for all patients and on overcoming high relapse rates in patients with high risk disease.