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. 2025 Apr 18;15(1):13402.
doi: 10.1038/s41598-025-98059-z.

Maintenance therapy with Azacitidine for patients with myeloid malignancies after allogeneic hematopoietic stem cell transplantation

Linli Lu #  1 , Qian Cheng #  2 , Yuhan Yan  1 , Xin Chen  3 , Yishu Tang  4 , Xin Li  5
Affiliations

Maintenance therapy with Azacitidine for patients with myeloid malignancies after allogeneic hematopoietic stem cell transplantation

Linli Lu et al. Sci Rep. .

Abstract

Disease relapse is a major cause of treatment failure after allogeneic haematopoietic stem cell transplantation (allo-HSCT) in patients with acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS). We retrospectively evaluated all patients (n = 145) with high-risk AML and MDS who underwent allo-HSCT between 2016 and 2022. Among them, 53 patients received maintenance therapy with azacitidine (AZA), and 10 of these patients received preemptive therapy. The rest of the 92 patients were in the control arm. The median follow-up time was 1215 days (103-3173 days). The median number of administered AZA cycles was 8. The 2-year relapse-free survival was 86.8% (46/53) in the AZA group compared with 76.1% (70/92) in the control group (P = 0.121). The 2-year overall survival in the AZA and control groups were 88.7% (47/53) and 82.6% (76/92), respectively (P = 0.326). Six patients (11.3%) in the AZA group experienced relapse, all of whom had a minimal residual disease (MRD)-positive status pre-HSCT. A total of 17 (32.1%) patients experienced grade 3-4 adverse events. In the preemptive therapy group, 70% (7/10) of the patients achieved complete remission after AZA treatment. AZA maintenance therapy following allo-HSCT is well tolerated and further follow-up and a larger group of patients will be necessary to confirm the potential to prevent relapse. The pre-transplant MRD status was identified as an important factor affecting the efficacy of AZA maintenance therapy.

Keywords: Acute myeloid leukemia; Allogeneic stem cell transplantation; Azacitidine; Minimal residual disease; Myelodysplastic syndromes.

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Conflict of interest statement

Declarations. Competing interests: The authors declare no competing interests. Ethics approval and consent to participate: All procedures were performed in compliance with relevant laws. This study was approved by the ethical committee of the Institute of the Third Xiangya Hospital of Central South University, ethical approval number was 23409, date June 5, 2024. Consent for publication: The privacy rights of patients have been observed, Informed consent was obtained from available subjects involved in this retrospective study, and that the patient(s) (or relative/guardian) consented to the publishing of all clinical data, and other data included in the manuscript.

Figures

Fig. 1
Fig. 1
Flowchart of the study participants.
Fig. 2
Fig. 2
Changes in the mean absolute counts of lymphocyte subsets and treg after azacitidine maintenance treatment. 2a, mean absolute counts of NK, CD4 + T and B lymphocyte; 2b, mean absolute counts of T lymphocyte and CD8 + T cells; 2c, mean absolute counts of treg cells.
Fig. 3
Fig. 3
Relapse free survival (3a), cumulative relapse rate(3b) and overall survival (3c). AZA maintenance did not significantly impact outcomes compared to control group.
Fig. 4
Fig. 4
Cumulative relapse rate after post-transplant azacitidine maintenance therapy in pre-transplant minimal residual disease positive patients.
Fig. 5
Fig. 5
Preemptive treatment and response.
See this image and copyright information in PMC

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