文章：

前瞻性、随机、比较性研究：氟达拉滨/白消安与氟达拉滨/白消安/全身照射预处理方案在髓系疾病中的对比分析

Prospective, Randomized, Comparative Study of Myeloablative Fludarabine/Busulfan and Fludarabine/Busulfan/Total Body Irradiation Conditioning in Myeloid Diseases

原文发布日期：28 March 2025

DOI: 10.3390/cancers17071140

类型: Article

开放获取: 是

英文摘要：

Background/Objectives: Allogeneic hematopoietic stem cell transplantation (alloSCT) is a curative treatment for myeloid diseases, yet relapse remains the major cause of post-transplant mortality. To reduce the risk of recurrence, we evaluated the addition of 400 cGy total body irradiation (TBI) to conditioning with fludarabine-busulfan (Flu/Bu4).Methods: In this prospective study, 46 patients with myeloid diseases were randomized to Flu/Bu4 or Flu/Bu4/TBI conditioning group. The Flu/Bu4 conditioning regimen consisted of fludarabine 40 mg/m2on days -6 to -3 followed by busulfan 130 mg/m2on days -6 to -3. Flu/Bu4/TBI conditioning regimen added 400 cGy TBI on day -1 to the FluBu4 regimen.Results: Among 34 acute myeloid leukemia (AML) patients, relapse was numerically lower in those who received Flu/Bu4/TBI (25%) versus Flu/Bu4 (44.4%) at three years (HR = 0.58, 95% CI 0.19 to 1.81,p= 0.35). Flu/Bu4/TBI appeared to increase the risk of non-relapse mortality (NRM) vs. Flu/Bu4 in AML patients at three years (25.0% versus 11.1%; HR = 2.11, 95% CI 0.51 to 8.83,p= 0.65). Overall survival (OS) was similar in AML patients undergoing conditioning with Flu/Bu4 (72.2%) versus Flu/Bu4/TBI (62.5%) at one year (p= 0.4).Conclusions: In conclusion, the addition of 400 cGy TBI to Flu/Bu4 reduced the risk of relapse but did not improve OS as a consequence of higher regimen-related mortality. Clinicaltrials.gov identifier: NCT01366612.

摘要翻译： 

背景/目的：异基因造血干细胞移植（alloSCT）是治疗髓系疾病的有效方法，但复发仍是移植后死亡的主要原因。为降低复发风险，本研究评估在氟达拉滨-白消安（Flu/Bu4）预处理方案基础上联合400 cGy全身照射（TBI）的效果。 方法：在这项前瞻性研究中，46例髓系疾病患者被随机分配至Flu/Bu4预处理组或Flu/Bu4/TBI预处理组。Flu/Bu4预处理方案为：第-6至-3天给予氟达拉滨40 mg/m²，随后在第-6至-3天给予白消安130 mg/m²。Flu/Bu4/TBI预处理方案则在Flu/Bu4方案基础上，于第-1天加用400 cGy TBI。 结果：在34例急性髓系白血病（AML）患者中，接受Flu/Bu4/TBI预处理的患者三年复发率（25%）较Flu/Bu4组（44.4%）呈降低趋势（HR=0.58，95% CI 0.19-1.81，p=0.35）。但Flu/Bu4/TBI方案可能增加AML患者三年非复发死亡率（NRM）（25.0% vs 11.1%；HR=2.11，95% CI 0.51-8.83，p=0.65）。两组AML患者一年总生存率（OS）相近：Flu/Bu4组72.2% vs Flu/Bu4/TBI组62.5%（p=0.4）。 结论：在Flu/Bu4预处理方案中联合400 cGy TBI虽可降低复发风险，但因方案相关死亡率升高，未能改善总生存率。临床试验注册号：NCT01366612。

原文链接：

Prospective, Randomized, Comparative Study of Myeloablative Fludarabine/Busulfan and Fludarabine/Busulfan/Total Body Irradiation Conditioning in Myeloid Diseases