{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T16:04:55Z","timestamp":1790697895399,"version":"4.1.0"},"reference-count":24,"publisher":"MDPI AG","issue":"19","license":[{"start":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T00:00:00Z","timestamp":1790640000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82570203"],"award-info":[{"award-number":["82570203"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"award":["82570203"],"award-info":[{"award-number":["82570203"]}],"id":[{"id":"https:\/\/ror.org\/01h0zpd94","id-type":"ROR","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Clin. Med.","JCM"],"abstract":"<jats:p>Background: Mutations of the FLT3 gene, particularly internal tandem duplication (FLT3-ITD), define a biologically aggressive AML subtype with historically poor outcomes. Here we retrospectively assessed the clinical performance and tolerability of sorafenib with venetoclax plus azacitidine (VA) as front-line therapy in patients with newly diagnosed FLT3-ITD-mutated AML. Methods: Forty-five consecutive patients who received sorafenib plus VA induction were retrospectively analyzed. Post-remission therapy comprised sorafenib-based chemotherapy consolidation, continued sorafenib-VA, or allogeneic hematopoietic stem cell transplantation (allo-HSCT) after consolidation therapy according to clinical indication and guidelines. Composite complete remission (CRc), measurable residual disease (MRD), overall survival (OS), and relapse-free survival (RFS) were assessed. Results: The CR\/CRi rate was 95.6% (43\/45), and the FCM-MRD negativity rate after induction was 90.7%. At a median follow-up of 20.3 months, the 2-year OS and RFS of the entire cohort were 65.3% and 57.3%, respectively. The relapse rate in the patients receiving post-remission allo-HSCT after consolidation therapy was lower. Continued sorafenib-VA achieved a 2-year OS of 57.1% and a 2-year RFS of 64%. Multivariable analysis identified chemotherapy-based post-remission therapy (p = 0.016) and detectable MRD after the second consolidation cycle (p = 0.004) as independent adverse prognostic factors for RFS. No treatment-related mortality occurred. Conclusions: Sorafenib plus VA is a cost-effective, tolerable induction regimen achieving high remission and MRD negativity rates in FLT3-ITD-mutant AML. For transplant-eligible patients, allo-HSCT significantly improves long-term survival; for transplant-ineligible patients, continued sorafenib-VA yields sustained benefit.<\/jats:p>","DOI":"10.3390\/jcm15197576","type":"journal-article","created":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T15:14:37Z","timestamp":1790694877000},"page":"7576","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Efficacy of Sorafenib Combined with Venetoclax and Azacitidine in Newly Diagnosed FLT3-ITD-Mutant Acute Myeloid Leukemia: Remission Efficacy and Prognostic Influence of Post-Remission Consolidation Therapy"],"prefix":"10.3390","volume":"15","author":[{"given":"Qi","family":"Chen","sequence":"first","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"formal-analysis","vocabulary":"credit"},{"role":"writing-original-draft","vocabulary":"credit"},{"role":"methodology","vocabulary":"credit"}]},{"given":"Wenbing","family":"Duan","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"investigation","vocabulary":"credit"},{"role":"data-curation","vocabulary":"credit"}]},{"given":"Jinsong","family":"Jia","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"data-curation","vocabulary":"credit"}]},{"given":"Jing","family":"Wang","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"data-curation","vocabulary":"credit"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7749-8087","authenticated-orcid":false,"given":"Xiaosu","family":"Zhao","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"methodology","vocabulary":"credit"},{"role":"funding-acquisition","vocabulary":"credit"}]},{"given":"Guorui","family":"Ruan","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"methodology","vocabulary":"credit"},{"role":"funding-acquisition","vocabulary":"credit"}]},{"given":"Hongxia","family":"Shi","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"methodology","vocabulary":"credit"}]},{"given":"Yingjun","family":"Chang","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"methodology","vocabulary":"credit"}]},{"given":"Xiaojun","family":"Huang","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"},{"name":"Peking-Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, Peking University, Beijing 100871, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"conceptualization","vocabulary":"credit"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1182-8132","authenticated-orcid":false,"given":"Hao","family":"Jiang","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People\u2019s Hospital, Peking University, Beijing 100044, China"}],"role":[{"vocabulary":"crossref","role":"author"},{"role":"conceptualization","vocabulary":"credit"},{"role":"supervision","vocabulary":"credit"},{"role":"funding-acquisition","vocabulary":"credit"},{"role":"investigation","vocabulary":"credit"}]}],"member":"1968","published-online":{"date-parts":[[2026,9,29]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"4372","DOI":"10.1182\/blood-2002-05-1440","article-title":"Prognostic significance of activating FLT3 mutations in younger adults (16 to 60 years) with acute myeloid leukemia and normal cytogenetics: A study of the AML Study Group Ulm","volume":"100","author":"Frohling","year":"2002","journal-title":"Blood"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"3622","DOI":"10.1182\/blood-2010-05-283648","article-title":"FLT3 internal tandem duplication associates with adverse outcome and gene- and microRNA-expression signatures in patients 60 years of age or older with primary cytogenetically 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