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However, no study has directly compared combination regimens. In this retrospective cohort study, 59 patients (AML\u2009=\u200956, MDS\u2009=\u20093) received AZA-based maintenance post-allogeneic hematopoietic stem cell transplantation (allo-HSCT), as monotherapy (n\u2009=\u200933), AZA plus interferon-\u03b1\u00a0(IFN-\u03b1) (n\u2009=\u200915), or AZA plus targeted agents (n\u2009=\u200911). At the median 31-month follow-up, the overall relapse rate was 10.2% (6\/59), with comparable rates across groups (AZA: 9.1%, AZA\u2009+\u2009IFN-\u03b1: 13.3%, AZA\u2009+\u2009targeted: 9.1%) (P\u2009=\u20090.850). Three 3-year relapse-free survival (89.4%; 95%CI 84.7\u201394.1%) and overall survival (84.4%; 95%CI 78.9\u201389.9%) rates did not differ significantly between monotherapy and combination regimens (RFS P\u2009=\u20090.975; OS P\u2009=\u20090.770). Overall adverse event rates showed no statistical difference (P\u2009&gt;\u20090.05), although febrile reactions were more common with IFN-\u03b1 combination regimens (P\u2009&lt;\u20090.001). These findings demonstrate that AZA monotherapy has non-inferior efficacy compared with combination regimens and has a favorable toxicity profile, establishing it as a viable backbone for maintenance therapy in MRD-negative patients. Biomarker-driven combinations warrant prospective validation.<\/jats:p>","DOI":"10.1007\/s12185-025-04097-8","type":"journal-article","created":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T16:39:30Z","timestamp":1762879170000},"page":"270-278","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Azacitidine monotherapy versus combination regimens as post-HSCT maintenance therapy in high-risk myeloid malignancies: a retrospective cohort study"],"prefix":"10.1007","volume":"123","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8086-7189","authenticated-orcid":false,"given":"Ying","family":"Shen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pengyu","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aili","family":"He","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianli","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jie","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wanhong","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liufang","family":"Gu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bo","family":"Lei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xueying","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0895-7750","authenticated-orcid":false,"given":"Yun","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,11,11]]},"reference":[{"issue":"11","key":"4097_CR1","doi-asserted-by":"publisher","first-page":"1921","DOI":"10.1038\/s41591-021-01521-4","volume":"27","author":"A Niroula","year":"2021","unstructured":"Niroula A, Sekar A, Murakami MA, Trinder M, Agrawal M, Wong WJ, et al. 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