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 Marrow Transplant Clinical Trials Network #1102 study rationale, design, and methods. Biol Blood Marrow Transplant 2014 Oct;20(10):1566-72
Date
06/28/2014Pubmed ID
24972249Pubmed Central ID
PMC4169902DOI
10.1016/j.bbmt.2014.06.010Scopus ID
2-s2.0-84912532203 (requires institutional sign-in at Scopus site) 14 CitationsAbstract
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.
Author List
Saber W, Le Rademacher J, Sekeres M, Logan B, Lewis M, Mendizabal A, Leifer E, Appelbaum FR, Horowitz MM, Nakamura R, Cutler CSAuthors
Mary M. Horowitz MD, MS Professor in the Medicine department at Medical College of WisconsinBrent R. Logan PhD Director, Professor in the Data Science Institute department at Medical College of Wisconsin
Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin
MESH terms used to index this publication - Major topics in bold
Age FactorsAged
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