自体干细胞移植治疗首次缓解期的成人急性髓细胞白血病——白消安与美法仑联合方案对比白消安与环磷酰胺联合方案疗效更佳：欧洲血液与骨髓移植学会（EBMT）急性白血病工作组的一项回顾性研究
Autologous stem cell transplantation for adult acute myelocytic leukemia in first remission-Better outcomes after busulfan and melphalan compared with busulfan and cyclophosphamide: A retrospective study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation (EBMT).
作者信息
Gorin Norbert-Claude, Labopin Myriam, Czerw Tomasz, Pabst Thomas, Blaise Didier, Dumas Pierre-Yves, Nemet Damir, Arcese William, Trisolini Silvia Maria, Wu Depei, Huynh Anne, Zuckerman Tsila, Meijer Ellen, Cagirgan Seckin, Cornelissen Jan, Houhou Mohamed, Polge Emmanuelle, Mohty Mohamad, Nagler Arnon
机构信息
Department of Hematology and Cellular Therapy, European Society for Blood and Marrow Transplantation (EBMT), National Institute of Health and Medical Research (INSERM) Unit 938, St. Antoine Public Assistance Hospital, Pierre and Marie Curie University, Paris, France.
EBMT Office, INSERM Unit 938, St. Antoine Public Assistance Hospital, Pierre and Marie Curie University, Paris, France.
出版信息
Cancer. 2017 Mar 1;123(5):824-831. doi: 10.1002/cncr.30400. Epub 2016 Dec 1.
BACKGROUND
Autologous stem cell transplantation (ASCT) for adult acute myelogenous leukemia (AML) is a valid therapeutic option for patients with good-risk and intermediate-risk disease. The authors used the registry of the European Society for Blood and Marrow Transplantation to compare combined busulfan and melphalan (BUMEL) with combined busulfan and cyclophosphamide (BUCY) before transplantation.
METHODS
From 2005 to 2013, 853 patients with available cytogenetics underwent ASCT in first remission, including 257 after receiving BUMEL and 596 after receiving BUCY. The proportion of patients with good-risk AML was lower in those who received BUMEL (14% vs 20%; P = .02). More patients who received BUMEL underwent autograft in molecular remission (89% vs 78%; P = .02). Three years after transplantation, the relapse incidence (RI) was 48.7%, the leukemia-free survival (LFS) rate was 47.7%, the overall survival (OS) rate was 66.2%, and the nonrelapse mortality (NRM) rate was 3.6%.
RESULTS
Patients who underwent an autograft after receiving BUMEL fared better than those who underwent an autograft after receiving BUCY with a lower RI (39.5% vs 52.2%; hazard ratio [HR], 0.65; 95% confidence interval [CI], 0.49-0.87; P = .003) a better LFS (55.4% vs 44.6%; HR, 0.69; 95% CI, 0.53-0.89; P = .005), and a better OS (73.8% vs 63%; HR, 0.62; 95% CI, 0.47-0.82; P = .0007). There was no difference in the NRM rate (BUMEL vs BUCY, 4.5% vs 3.2%, respectively). Among 74 patients in the BUMEL group and 187 in the BUCY group who underwent autograft in molecular remission, the RI was 30% versus 51%, respectively (univariate analysis; P = .01), and the LFS rate was 66% versus 47%, respectively (univariate analysis; P = .03).
CONCLUSIONS
In patients with AML in first complete remission who undergo ASCT, the BUMEL combination is a better preparative regimen. Cancer 2017;123:824-31. © 2016 American Cancer Society.
背景
自体干细胞移植（ASCT）治疗成人急性髓系白血病（AML）对于低危和中危疾病患者是一种有效的治疗选择。作者利用欧洲血液和骨髓移植学会的登记数据比较了移植前白消安联合美法仑（BUMEL）与白消安联合环磷酰胺（BUCY）的疗效。
方法
2005年至2013年，853例有可用细胞遗传学数据的患者在首次缓解期接受了ASCT，其中257例接受BUMEL治疗，596例接受BUCY治疗。接受BUMEL治疗的患者中低危AML患者的比例较低（14%对20%；P = 0.02）。更多接受BUMEL治疗的患者在分子缓解期进行了自体移植（89%对78%；P = 0.02）。移植后三年，复发率（RI）为48.7%，无白血病生存率（LFS）为47.7%，总生存率（OS）为66.2%，非复发死亡率（NRM）为3.6%。
结果
接受BUMEL治疗后进行自体移植的患者比接受BUCY治疗后进行自体移植的患者预后更好，RI更低（39.5%对52.2%；风险比[HR]，0.65；95%置信区间[CI]，0.49 - 0.87；P = 0.003），LFS更好（55.4%对44.6%；HR，0.69；95% CI，0.53 - 0.89；P = 0.005），OS也更好（73.8%对63%；HR，0.62；95% CI，0.47 - 0.82；P = 0.0007）。NRM率无差异（BUMEL组对BUCY组分别为4.5%对3.2%）。在BUMEL组的74例和BUCY组的187例在分子缓解期进行自体移植的患者中，RI分别为30%和51%（单因素分析；P = 0.01），LFS率分别为66%和47%（单因素分析；P = 0.03）。
结论
在首次完全缓解期接受ASCT的AML患者中，BUMEL联合方案是更好的预处理方案。《癌症》2017年；123：824 - 31。©2016美国癌症协会
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