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Pre-transplant azacitidine does not improve survival in allogeneic HSCT for higher-risk MDS

Bone Marrow Transplantation (2026)Cite this article

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To the Editor:

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative therapy for myelodysplastic syndromes (MDS) and optimising patients for transplant is critical to achieving the best outcomes [1, 2]. Hypomethylating agents (HMAs), particularly azacitidine, are used as a bridging strategy with the aim of achieving disease control and limiting leukemic transformation. The role of pre-transplant azacitidine in higher-risk transplant-eligible MDS has remained uncertain and recent studies comparing azacitidine to upfront transplant have not demonstrated a clear survival benefit [3,4,5]. We undertook a retrospective multicentre study across five Australian transplant centres to evaluate the association of pre-transplant azacitidine with survival after allo-HSCT. This study was approved by the institutional review board of Melbourne Health with a waiver of informed consent (2023.094).

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Fig. 1: Outcomes following allogeneic haematopoietic stem cell transplantation in higher-risk MDS with pre-transplant azacitidine.
The alternative text for this image may have been generated using AI.

Data availability

The datasets generated and analyzed during this study can be made available upon reasonable request to the corresponding author. Decisions regarding data sharing will be made on a case-by-case basis, considering data protection and other applicable regulations.

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Authors and Affiliations

  1. Clinical Hematology, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC, Australia

    Miles Horton, Mark R. Dowling, Lynette C. Y. Chee, David S. Ritchie & Ashish Bajel

  2. The Walter and Eliza Hall Institute of Medical Research, Parkville, VIC, Australia

    Miles Horton

  3. Cancer Care Services, Royal Brisbane and Women’s Hospital, Brisbane, QLD, Australia

    Becky Leung, Ricky Nelles, Stewart Hunt, Ashleigh P. Scott & Glen A. Kennedy

  4. Griffith University School of Medicine, Southport, QLD, Australia

    Becky Leung

  5. Faculty of Medicine, University of Queensland, Brisbane, QLD, Australia

    Ricky Nelles & Ashleigh P. Scott

  6. Westmead Hospital, Westmead, NSW, Australia

    Matthew Rubic & Abir Bhattacharya

  7. Haematology Department, Fiona Stanley Hospital, Murdoch, WA, Australia

    Christopher McKinnely & Duncan Purtill

  8. Royal Adelaide Hospital, Adelaide, SA, Australia

    Alia Cibich & Devendra Hiwase

  9. Department of Medicine, University of Melbourne, Melbourne, VIC, Australia

    Lynette C. Y. Chee, David S. Ritchie & Ashish Bajel

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  1. Miles Horton
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Contributions

MH: data collection, data analysis and interpretation, manuscript drafting and accessed and verified the underlying data reported in the manuscript. MRD: concept and design, statistical analysis and interpretation of data. AB: concept and design, data analysis and interpretation, manuscript drafting and project administration. BL, RN, MR, CM, AC, SH, LCYC, APS, DSR, DH, DP, ABh and GAK: data collection, analysis and interpretation of data and manuscript drafting. All authors reviewed, revised and approved the final manuscript for submission.

Corresponding author

Correspondence to Ashish Bajel.

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Competing interests

MRD has patents and royalties with AbbVie and received consultancy fees from Kite/Gilead and Novartis. LCYC received honoraria and served on advisory boards for Novartis and Otsuka and received consultancy fees and honoraria from Keros Therapeutics. DSR received research funding from BMS, Novartis and Takeda, received consultancy fees from Novartis and served on advisory boards for Novartis, Takeda and Amgen. DH received honoraria from AbbVie, Otsuka and Astellas Pharma. AB received honoraria and served on advisory boards for AbbVie, Amgen and Astellas; received honoraria from Novartis, Pfizer and Takeda; and served on advisory boards for Glaxo-Smith-Kline. The remaining authors declare no competing financial interests.

Ethics approval and informed consent

This study was approved by the institutional review board of Melbourne Health with a waiver of informed consent (2023.094) with governance approval of the review boards of participating sites. This work was conducted under the Declaration of Helsinki and Australian data regulations.

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Horton, M., Leung, B., Nelles, R. et al. Pre-transplant azacitidine does not improve survival in allogeneic HSCT for higher-risk MDS. Bone Marrow Transplant (2026). https://doi.org/10.1038/s41409-026-02874-5

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