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Bone Marrow Transplant. 2019 May 14. doi: 10.1038/s41409-019-0536-y. [Epub ahead of print]

Prospective phase II study of prophylactic low-dose azacitidine and donor lymphocyte infusions following allogeneic hematopoietic stem cell transplantation for high-risk acute myeloid leukemia and myelodysplastic syndrome.

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Hematology Department, Nantes University Hospital, Nantes, France.
Cancer Research Center Nantes-Angers, INSERM U1232, Nantes, France.
Hematology Department, AP-HP, Saint-Antoine Hospital, Paris, France.
INSERM UMR 938 and Pierre et Marie Curie University, Paris, France.
Bone Marrow Transplantation Unit, Lille University Hospital, Lille, France.
Hematology and Cell Therapy Department, Bordeaux University Hospital, Pessac, France.
Hematology Department, Institut Universitaire du Cancer Toulouse - Oncopole, Toulouse, France.
Hematology Department, Nantes University Hospital, Nantes, France.
Cancer Research Center Nantes-Angers, INSERM U1232, Nantes, France.
Lille Inflammation Research Center, INSERM U995, University of Lille, Nantes, France.


Thirty patients, with high-risk acute myeloid leukemia (AML, n = 20) or myelodysplastic syndrome (MDS, n = 10), were enrolled in a phase II trial entailing prophylactic post-transplant azacitidine (AZA) plus escalated doses of donor lymphocyte infusion (DLI). The median number of AZA cycles was 5 (1-12) with 10 patients (33%) completing the 12 projected cycles. DLI were performed in 17 patients: 5 received one DLI, 2 received 2 DLI and 8 received 3 infusions. AZA was well tolerated, but discontinued in 20 patients primarily due to graft-versus-host disease (GvHD) and relapse. The cumulative incidence (CI) of grade 1-3 acute GvHD was 31.5% and the chronic GvHD CI was 53% at 2 years. At a median follow-up of 49 months (27-63), 18 patients are alive. The overall and disease-free survivals are 65.5% (CI 95% = 48.2-82.8) at 2 years. Cause of death was mainly relapse for 9 patients. The median time to relapse was 7 months (2.5-58) and the cumulative incidence of relapse at 2 years was 27.6% (CI 95% = 12.8-44.6). These results confirm that AZA is well tolerated as a prophylactic treatment to reduce the risk of post-transplantation relapse and compared favorably to those of patients who receive no post-transplant maintenance.


azacitidine; donor lymphocyte infusion; relapse prevention


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