芦可替尼与地西他滨联合白消安-环磷酰胺预处理方案用于高危急性髓系白血病或骨髓增生异常综合征患者的复发预防
Ruxolitinib and decitabine plus a busulfan-cyclophosphamide conditioning regimen for relapse prophylaxis in patients with high-risk acute myeloid leukemia or myelodysplastic syndromes.
作者信息
Wei Yujun, Luan Songhua, Wang Lu, Wang Lili, Li Fei, Jin Xiangshu, Yang Ruoling, Qian Kun, Peng Bo, Tang Jingwen, Zhang Haoyang, Dou Liping, Liu Daihong
机构信息
School of Medicine, Nankai University, Tianjin, China.
The Chief Department of Hematology, the Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, China.
出版信息
Front Immunol. 2025 Aug 18;16:1586512. doi: 10.3389/fimmu.2025.1586512. eCollection 2025.
PURPOSE
Relapse remains the leading cause of treatment failure in high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome-IB (MDS-IB) patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ruxolitinib has demonstrated antileukemic activity , and decitabine has been found to be tolerable when combined with modified busulfan-cyclophosphamide (mBu/Cy) conditioning regimen. Here, we investigated the efficacy of ruxolitinib and decitabine plus a mBu/Cy conditioning regimen (Rux-Dec-mBu/Cy) in reducing relapse in high-risk AML/MDS patients ().
PATIENTS AND METHODS
This prospective investigational study enrolled 58 patients between May 2020 and July 2023. These patients had either a relapsed/refractory status, remission status with adverse genetic abnormalities or positive measurable residual disease (MRD+) prior to conditioning. Ruxolitinib (days -15 to -1) and decitabine (days -15 to -10) were administered, followed by mBu/Cy conditioning. The outcomes of a historical cohort of 58 patients (matched 1:1) who received mBu/Cy are described for reference.
RESULTS
All 58 patients achieved engraftment. With a median follow-up of 967 (464-1597) days, the 2-year cumulative incidence of relapse was 19.0%. The probabilities of 2-year overall survival (OS), disease-free survival (DFS) and graft-versus-host disease-free, relapse-free survival (GRFS) were 70.3%, 70.6% and 65.2%, respectively. The cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD) was 44.1%. The most common grade ≥3 adverse event was oropharyngeal mucositis (8.6%, n=5). Within 6 months post-transplantation, the cumulative incidence of cytomegalovirus (CMV) reactivation was 34.5%, and that of Epstein-Barr virus (EBV) reactivation was 62.1%.
CONCLUSIONS
This investigational study revealed that the Rux-Dec-mBu/Cy conditioning was tolerable and reduced relapse in high-risk AML/MDS patients.
目的
复发仍然是高危急性髓系白血病（AML）或骨髓增生异常综合征-IB（MDS-IB）患者异基因造血干细胞移植（allo-HSCT）后治疗失败的主要原因。芦可替尼已显示出抗白血病活性，并且当与改良白消安-环磷酰胺（mBu/Cy）预处理方案联合使用时，地西他滨已被证明是可耐受的。在此，我们研究了芦可替尼和地西他滨联合mBu/Cy预处理方案（Rux-Dec-mBu/Cy）在降低高危AML/MDS患者复发率方面的疗效。
患者和方法
这项前瞻性研究在2020年5月至2023年7月期间招募了58名患者。这些患者在预处理前处于复发/难治状态、缓解状态但伴有不良遗传异常或可测量残留病阳性（MRD+）。给予芦可替尼（第-15天至-1天）和地西他滨（第-15天至-10天），随后进行mBu/Cy预处理。描述了一组接受mBu/Cy预处理的58例历史队列患者（1:1匹配）的结果以供参考。
结果
所有58例患者均实现造血重建。中位随访967（范围464 - 1597）天，2年累积复发率为19.0%。2年总生存（OS）、无病生存（DFS）以及无移植物抗宿主病、无复发生存（GRFS）的概率分别为70.3%、70.6%和65.2%。II-IV级急性移植物抗宿主病（aGVHD）的累积发生率为44.1%。最常见的≥3级不良事件是口腔黏膜炎（8.6%，n = 5）。移植后6个月内，巨细胞病毒（CMV）再激活的累积发生率为34.5%，EB病毒（EBV）再激活的累积发生率为62.1%。
结论
这项研究表明，Rux-Dec-mBu/Cy预处理方案在高危AML/MDS患者中是可耐受的，并降低了复发率。
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