Junjie Cao, Xianxu Zhuang, Danjie Luo, Renzhi Pei, Ying Lu, Dong Chen, Shuangyue Li, Xiaohong Du, Xuhui Liu
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This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older.</p><p><strong>Methods: </strong>We conducted a retrospective study on AML patients aged 55 to 70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haplo-HSCT based on disease risk degree, measurable residual disease status and patient's preference.</p><p><strong>Results: </strong>Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year over survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (P<0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% vs 69.3% (P = 0.367) for OS, and 74.5% vs 69.7% (P = 0.473) for RFS. High-risk ELN stratification and the presence of ≥4 gene mutations were associated with lower OS and RFS in both univariate and multivariate analysis.</p><p><strong>Conclusions: </strong>AML patients over 55 years of age who received haplo-HSCT after two courses of VEN/AZA therapy had outcomes similar to those who received haplo-HSCT after intensive chemotherapy, suggesting that two courses of VEN/AZA therapy as a bridge to halpo-HSCT are feasible for patients over 55 years old.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-18"},"PeriodicalIF":1.7000,"publicationDate":"2024-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Efficacy of haploidentical allogeneic hematopoietic cell transplantation following two courses of venetoclax and azacytidine therapy in patients over 55 years old with acute myelogenous leukemia.\",\"authors\":\"Junjie Cao, Xianxu Zhuang, Danjie Luo, Renzhi Pei, Ying Lu, Dong Chen, Shuangyue Li, Xiaohong Du, Xuhui Liu\",\"doi\":\"10.1159/000542034\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation is a promising potential cure for high-risk AML, as VEN-based therapies have a worse prognosis in elderly patients. This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older.</p><p><strong>Methods: </strong>We conducted a retrospective study on AML patients aged 55 to 70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haplo-HSCT based on disease risk degree, measurable residual disease status and patient's preference.</p><p><strong>Results: </strong>Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year over survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (P<0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% vs 69.3% (P = 0.367) for OS, and 74.5% vs 69.7% (P = 0.473) for RFS. 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引用次数: 0
摘要
简介venetoclax（VEN）和氮杂胞嘧啶（AZA）的联合治疗已证明可使老年急性髓性白血病（AML）患者获得快速有效的缓解。异基因造血干细胞移植是治疗高风险急性髓细胞白血病的一种很有前景的潜在疗法，因为基于VEN的疗法在老年患者中的预后较差。本研究旨在评估55岁及以上急性髓细胞性白血病患者在接受两个疗程的VEN和AZA治疗后进行序贯单倍体造血干细胞移植的疗效：我们对55岁至70岁的AML患者进行了一项回顾性研究，这些患者接受了强化化疗或两个疗程的VEN/AZA治疗，随后根据疾病风险程度、可测量的残留疾病状态和患者的偏好进行了单倍体造血干细胞移植：2019年1月至2023年12月期间，141名新确诊的急性髓细胞白血病患者接受了强化化疗或VEN/AZA疗法的初始治疗。其中，64名患者接受了单倍体造血干细胞移植，77名患者没有接受。接受单倍体-HSCT的患者的1年生存率（OS）和无复发生存率（RFS）明显高于未接受单倍体-HSCT的患者（P<0.05）。在接受移植的患者中，VEN/AZA组和强化化疗组的1年OS和RFS无明显差异：OS为76.3% vs 69.3% (P = 0.367)，RFS为74.5% vs 69.7% (P = 0.473)。在单变量和多变量分析中，高风险ELN分层和存在≥4基因突变与较低的OS和RFS有关：结论：55岁以上的急性髓细胞白血病患者在接受两个疗程的VEN/AZA治疗后接受单倍体-HSCT治疗，其结果与强化化疗后接受单倍体-HSCT治疗的患者相似，这表明55岁以上的患者可以接受两个疗程的VEN/AZA治疗作为通往halpo-HSCT的桥梁。
Efficacy of haploidentical allogeneic hematopoietic cell transplantation following two courses of venetoclax and azacytidine therapy in patients over 55 years old with acute myelogenous leukemia.
Introduction: The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation is a promising potential cure for high-risk AML, as VEN-based therapies have a worse prognosis in elderly patients. This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older.
Methods: We conducted a retrospective study on AML patients aged 55 to 70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haplo-HSCT based on disease risk degree, measurable residual disease status and patient's preference.
Results: Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year over survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (P<0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% vs 69.3% (P = 0.367) for OS, and 74.5% vs 69.7% (P = 0.473) for RFS. High-risk ELN stratification and the presence of ≥4 gene mutations were associated with lower OS and RFS in both univariate and multivariate analysis.
Conclusions: AML patients over 55 years of age who received haplo-HSCT after two courses of VEN/AZA therapy had outcomes similar to those who received haplo-HSCT after intensive chemotherapy, suggesting that two courses of VEN/AZA therapy as a bridge to halpo-HSCT are feasible for patients over 55 years old.
期刊介绍：
''Acta Haematologica'' is a well-established and internationally recognized clinically-oriented journal featuring balanced, wide-ranging coverage of current hematology research. A wealth of information on such problems as anemia, leukemia, lymphoma, multiple myeloma, hereditary disorders, blood coagulation, growth factors, hematopoiesis and differentiation is contained in first-rate basic and clinical papers some of which are accompanied by editorial comments by eminent experts. These are supplemented by short state-of-the-art communications, reviews and correspondence as well as occasional special issues devoted to ‘hot topics’ in hematology. These will keep the practicing hematologist well informed of the new developments in the field.
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