At this year's American Society of Hematology Annual Meeting, I presented the results of the Blood and Marrow Transplant Clinical Trial Networks trial studying allogeneic hematopoietic cell transplantation (HCT) for older patients with advanced myelodysplastic syndrome (MDS). Early HCT for older patients with MDS is infrequently offered, since the relative benefits of HCT over non-HCT therapy have not been well defined in this patient group. In this study, patients aged 50-75 with higher risk MDS who were candidates for reduced-intensity conditioning (RIC) HCT were enrolled and assigned to the 'Donor' group if a suitable 8/8 HLA-matched donor (related or unrelated) was available, and to the 'No Donor' group if no match was available. Subjects were expected to undergo HCT in the Donor group, and to receive standard non-HCT therapy in the No Donor group. A total of 384 subjects (Donor n=260, No Donor n=124) were enrolled at 34 centers.
Overall survival at 3 years following treatment assignment in the Donor arm was significantly higher than the No Donor arm (47.9% vs. 26.6%, p=0.0001).
Similarly, leukemia-free survival was higher in the Donor arm (35.8% vs 20.6%, p=0.003), and this benefit was seen across all patient subgroups. Importantly, there were no clinically significant differences in quality of life between Donor and No Donor arms, an important finding as HCT is often not offered because of a fear of loss of quality of life.
Due to the striking differences in outcomes between the two treatment groups, HCT should be included as an integral part of MDS management plans in fit older adults with higher risk MDS. Importantly, since identifying a suitable donor for older adults may require several weeks, early referral to a transplant center is strongly recommended to begin the donor search process and to begin conversations on the role of HCT for individual patients.