Žučenka Andrius, Tamutytė Milvydė
Department of Hematology and Oncology, Institute of Clinical Medicine, Faculty of Medicine, Vilnius University, Vilnius, Lithuania.
Front Oncol. 2025 Mar 27;15:1530852. doi: 10.3389/fonc.2025.1530852. eCollection 2025.
The prognosis of r AML is poor, with a 5-year overall survival (OS) rate of less than 10%. This is mostly attributable to the low efficacy of all available therapies and high relapse rates even after allogeneic stem cell transplantation (alloSCT), which remains the only curative approach. We report upfront sequential alloSCT with venetoclax-based preconditioning as a safe and effective treatment for two newly diagnosed and fit r AML patients. The sequential alloSCT regimen consisted of triple therapy: preconditioning with decitabine and venetoclax on days 1-5 followed by FLAG-Ida and venetoclax on days 6-10. One or 3 days after preconditioning, the patients underwent busulfan-based myeloablative conditioning and HLA haploidentical or matched related donor stem cell infusion. One month after alloSCT, timely engraftment and complete remission were achieved. At the last follow-up, both patients were in good health and in MRD-negative complete remissions after 11 and 17 months after alloSCT, respectively. The safety and efficacy of upfront sequential alloSCT indicate the need to evaluate this approach for adverse risk of AML in clinical trials.
复发/难治性急性髓系白血病(r AML)的预后较差,5年总生存率(OS)低于10%。这主要归因于所有现有治疗方法疗效欠佳,即便在进行异基因干细胞移植(alloSCT)后复发率仍很高,而异基因干细胞移植仍是唯一的治愈方法。我们报告了采用基于维奈克拉的预处理方案进行早期序贯异基因干细胞移植,作为对两名新诊断且身体状况良好的r AML患者的一种安全有效的治疗方法。序贯异基因干细胞移植方案包括三联疗法:第1 - 5天使用地西他滨和维奈克拉进行预处理,随后在第6 - 10天使用FLAG-伊达比星和维奈克拉。预处理后1天或3天,患者接受基于白消安的清髓性预处理以及HLA单倍型相合或匹配的相关供者干细胞输注。异基因干细胞移植后1个月,实现了及时植入和完全缓解。在最后一次随访时,两名患者均身体健康,分别在异基因干细胞移植后11个月和17个月处于微小残留病(MRD)阴性的完全缓解状态。早期序贯异基因干细胞移植的安全性和有效性表明,有必要在临床试验中评估这种方法对AML不良风险的治疗效果。