Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome.
2021 Oct 20
Journal Article
Authors:
Nakamura, R.;
Saber, W.;
Martens, M.J.;
Ramirez, A.;
Scott, B.;
Oran, B.;
Leifer, E.;
Tamari, R.;
Mishra, A.;
Maziarz, R.T.;
McGuirk, J.;
Westervelt, P.;
Vasu, S.;
Patnaik, M.;
Kamble, R.;
Forman, S.J.;
Sekeres, M.A.;
Appelbaum, F.;
Mendizabal, A.;
Logan, B.;
Horowitz, M.;
Cutler, C.
Secondary:
J Clin Oncol
Volume:
39
Pagination:
3328-3339
Issue:
30
PMID:
34106753
URL:
https://pubmed.ncbi.nlm.nih.gov/34106753/
Keywords:
Allogeneic hematopoietic cell transplantation (HCT); hypomethylating therapy; myelodysplastic syndromes (MDS)
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 ( = .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; = .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.
