摘要：Allogeneic hematopoetic stem-cell transplantation (SCT) is a curative treatment for a variety of hematological malignancies. There is a continuous search for novel condition- ing regimens that will reduce SCT-related toxicity while retaining maximal anti-malignan- cy effect. Treosulfan (Treo) was initially used in the treatment of certain solid tumors. Pre- clinical studies showed that in contrast to Busulfan it has an effect on both committed and non- committed hematopoietic stem cells. It also has stronger immunosuppressive characteristics that make it suitable for conditioning regimens for allogeneic SCT includ- ing from cord blood and Haploidentical donors. Treo is also associated with a favorable toxicity profile with little extra-medullary toxicity. The combination of Fludarabine (Flu) and Treo was explored in several studies in patients not eligible for standard myeloabla- tive conditioning, and data are rapidly emerging. The Flu/Treo regimen is associated with consistent engraftment, limited non-relapse mortality, low rates of organ toxicity as well as acute and chronic graft versus host disease (GVHD). The regimen was also associated with low relapse rates of 5-30% depending on disease status at SCT. All to- gether resulted in favorable survival of 40-80%. Promising results were seen mainly in my- elodysplastic syndrome (survival of 36-70%) and myeloid leukemia in remission (60-70%). Randomized prospective studies will be needed to better define the role of Treosulfan- based regimens in SCT.
