Multicenter biologic assignment trial comparing reduced-intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50 to 75 with intermediate-2 and high-risk myelodysplastic syndrome: Blood and M

Publication Type
Journal Article
Year of Publication
Saber, Wael; Le Rademacher, Jennifer; Sekeres, Mikkael; Logan, Brent; Lewis, Moira; Mendizabal, Adam; Leifer, Eric; Appelbaum, Frederick R; Horowitz, Mary M; Nakamura, Ryotaro; Cutler, Corey S
Biol Blood Marrow Transplant
Date Published
2014 Oct
Age Factors; Aged; Antimetabolites, Antineoplastic; Cost-Benefit Analysis; DNA Methylation; Female; Hematopoietic Stem Cell Transplantation; Histocompatibility Testing; Humans; Intention to Treat Analysis; Male; Middle Aged; Myeloablative Agonists; Myelodysplastic Syndromes; prognosis; Prospective Studies; quality of life; Risk Factors; Survival Analysis; Transplantation Conditioning; Transplantation, Homologous; Unrelated Donors

The introduction of reduced-intensity conditioning (RIC) regimens made it possible to offer allogeneic hematopoietic cell transplantation (alloHCT) to older patients with myelodysplastic syndromes (MDS). However, the relative risks and benefits of alloHCT compared with novel nontransplant therapies continue to be the source of considerable uncertainty. We will perform a prospective biologic assignment trial to compare RIC alloHCT with nontransplant therapies based on donor availability. Primary outcome is 3-year overall survival. Secondary outcomes include leukemia-free survival, quality of life, and cost-effectiveness. Four hundred patients will be enrolled over roughly 3 years. Planned subgroup analyses will evaluate key biologic questions, such as the impact of age and response to hypomethylating agents on treatment effects. Findings from this study potentially may set a new standard of care for older MDS patients who are considered candidates for alloHCT.