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*

Publication Type
Journal Article
Year of Publication
2014
Authors
Saber, W; Le Rademacher, J; Sekeres, M; Logan, B; Lewis, M; Mendizabal, A; Leifer, E; Appelbaum, FR; Horowitz, MM; Nakamura, R; Cutler, CS
Secondary
Biol Blood Marrow Transplant
Volume
20
Start Page
1566
Pagination
1566-1572
Date Published
10/2014
Keywords
Biologic assignment; MDS; Transplantation
Abstract
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.
URL
http://www.ncbi.nlm.nih.gov/pubmed/24972249