Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation
Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation
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DOI：
10.1182/blood-2013-08-519009
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发表时间：
2013-12-05
期刊：
影响因子：
20.3
通讯作者：
Pasquini, Marcelo C.
中科院分区：
文献类型：
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作者：
Bredeson, Christopher;LeRademacher, Jennifer;Pasquini, Marcelo C.
我们进行了一项前瞻性队列研究，测试了骨髓恶性肿瘤患者中消融性静脉注射白消安（IV-BU）与消融性全身照射（TBI）方案的生存率非劣效性。共入组了1483例接受骨髓恶性肿瘤移植的患者（IV-BU，N = 1025; TBI，N = 458）。队列在年龄、性别、种族、性能评分、疾病和移植时的疾病分期方面相似。大多数患者患有急性髓系白血病（68% IV-BU，78% TBI）。移植物主要是来自HLA匹配的同胞（40%）或匹配良好的无关供体（48%）的外周血（77%）。IV-BU（相对风险0.82; 95%CI 0.68-0.98，P = 0.03）和TBI的两年生存概率（95%置信区间[CI]）分别为56%（95%CI，53%-60%）和48%（95%CI，43%-54%，P = 0.019）。相应的移植相关死亡率（TRM）为18%（95% CI，16%-21%）和19%（95% CI，15%-23%，P = 0.75），疾病进展为34%（95% CI，31%-37%）和39%（95% CI，34%-44%，P = 0.08）。IV-BU组肝静脉闭塞性疾病（VOD）的发生率为5%，TBI组为1%（P <0.001）。无进展生存期和移植物抗宿主病无差异。与TBI相比，IV-BU导致上级生存率，而复发或TRM风险未增加。这些结果支持使用清髓性IV-BU与基于TBI的预处理方案治疗骨髓恶性肿瘤。（血。2013;122（24）：3871-3878）
We conducted a prospective cohort study testing the noninferiority of survival of ablative intravenous busulfan (IV-BU) vs ablative total body irradiation (TBI)-based regimens in myeloid malignancies. A total of 1483 patients undergoing transplantation for myeloid malignancies (IV-BU, N = 1025; TBI, N = 458) were enrolled. Cohorts were similar with respect to age, gender, race, performance score, disease, and disease stage at transplantation. Most patients had acute myeloid leukemia (68% IV-BU, 78% TBI). Grafts were primarily peripheral blood (77%) from HLA-matched siblings (40%) or well-matched unrelated donors (48%). Two-year probabilities of survival (95% confidence interval [CI]), were 56% (95% CI, 53%-60%) and 48% (95% CI, 43%-54%, P = .019) for IV-BU (relative risk, 0.82; 95% CI, 0.68-0.98, P = .03) and TBI, respectively. Corresponding incidences of transplant-related mortality (TRM) were 18% (95% CI, 16%-21%) and 19% (95% CI, 15%-23%, P = .75) and disease progression were 34% (95% CI, 31%-37%) and 39% (95% CI, 34%-44%, P = .08). The incidence of hepatic veno-occlusive disease (VOD) was 5% for IV-BU and 1% with TBI (P < .001). There were no differences in progression-free survival and graft-versus-host disease. Compared with TBI, IV-BU resulted in superior survival with no increased risk for relapse or TRM. These results support the use of myeloablative IV-BU vs TBI-based conditioning regimens for treatment of myeloid malignancies. (Blood. 2013;122(24):3871-3878)