Faculty Collaboration Database

Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome. J Clin Oncol 2021 Oct 20;39(30):3328-3339

Date

06/10/2021

Pubmed ID

34106753

Pubmed Central ID

PMC8791814

DOI

10.1200/JCO.20.03380

Scopus ID

2-s2.0-85112086284 (requires institutional sign-in at Scopus site)   70 Citations

Abstract

PURPOSE: Allogeneic hematopoietic cell transplantation (HCT) is the only potentially curative therapy for myelodysplastic syndromes (MDS), although it is infrequently offered to older patients. The relative benefits of HCT over non-HCT therapy in older patients with higher-risk MDS have not been defined.

METHODS: We conducted a multicenter biologic assignment trial comparing reduced-intensity HCT to hypomethylating therapy or best supportive care in subjects 50-75 years of age with intermediate-2 or high-risk de novo MDS. The primary outcome was overall survival probability at 3 years. Between January 2014 and November 2018, we enrolled 384 subjects at 34 centers. Subjects were assigned to the Donor or No-Donor arms according to the availability of a matched donor within 90 days of study registration.

RESULTS: The median follow-up time for surviving subjects was 34.2 months (range: 2.3-38 months) in the Donor arm and 26.9 months (range: 2.4-37.2 months) in the No-Donor arm. In an intention-to-treat analysis, the adjusted overall survival rate at 3 years in the Donor arm was 47.9% (95% CI, 41.3 to 54.1) compared with 26.6% (95% CI, 18.4 to 35.6) in the No-Donor arm (P = .0001) with an absolute difference of 21.3% (95% CI, 10.2 to 31.8). Leukemia-free survival at 3 years was greater in the Donor arm (35.8%; 95% CI, 29.8 to 41.8) compared with the No-Donor arm (20.6%; 95% CI, 13.3 to 29.1; P = .003). The survival benefit was seen across all subgroups examined.

CONCLUSION: We observed a significant survival advantage in older subjects with higher-risk MDS who have a matched donor identified and underwent reduced-intensity HCT, when compared with those without a donor. HCT should be included as an integral part of MDS management plans in fit older adults with higher-risk MDS.

Author List

Nakamura R, Saber W, Martens MJ, Ramirez A, Scott B, Oran B, Leifer E, Tamari R, Mishra A, Maziarz RT, McGuirk J, Westervelt P, Vasu S, Patnaik M, Kamble R, Forman SJ, Sekeres MA, Appelbaum F, Mendizabal A, Logan B, Horowitz M, Cutler C

Authors

Mary M. Horowitz MD, MS Professor in the Medicine department at Medical College of Wisconsin
Brent R. Logan PhD Director, Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Michael Martens PhD Assistant Professor in the Institute for Health and Equity 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

Aged
Female
Follow-Up Studies
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Histocompatibility Testing
Humans
Intention to Treat Analysis
Leukemia
Male
Middle Aged
Myelodysplastic Syndromes
Quality of Life
Survival Rate
Tissue Donors
Transplantation Conditioning
Transplantation, Homologous
Treatment Outcome