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一项旨在确定急性白血病成年患者造血干细胞移植可行性的前瞻性注册研究:规划、预期与现实。

A prospective registration study to determine feasibility of hematopoietic SCT in adults with acute leukemia: planning, expectations and reality.

作者信息

Labopin M, Gorin N-C, Polge E, Socié G, Gurman G, Gluckman E, Jindra P, Poiré X, Schäfer-Eckart K, Ruutu T, Milone G, Arcese W, Mohty M, Rocha V

机构信息

1] Paris EBMT Data Coordination Office, Hospital Saint-Antoine, APHP, Université Pierre et Marie Curie UPMC and INSERM U 938, Paris, France [2] Department of Hematology and Cell Therapy, Hospital Saint-Antoine, Paris, France.

Paris EBMT Data Coordination Office, Hospital Saint-Antoine, APHP, Université Pierre et Marie Curie UPMC and INSERM U 938, Paris, France.

出版信息

Bone Marrow Transplant. 2014 Mar;49(3):376-81. doi: 10.1038/bmt.2013.178. Epub 2013 Nov 18.

DOI:10.1038/bmt.2013.178
PMID:24241579
Abstract

For adults with acute leukemia, it is important to know whether the therapeutic schemes initially planned were actually implemented. The European Group for Blood and Marrow transplantation Acute Leukemia Working Party prospectively followed 695 consecutive patients who were registered at the time of HLA typing. Of 304 patients with an available matched sibling donor (MSD), SCT was planned in 264, chemotherapy in 33 and autografting in 7. For the rest, an unrelated donor (UD) search was initiated in 198. Among these, 117 were transplanted, 114 received chemotherapy and 77 underwent autografting. Probabilities of receiving a planned treatment were 60 and 65% at 1 and 2 years, respectively. Patients scheduled to receive MSD SCT had an 82% probability, whereas those scheduled to undergo UD SCT had a 57% probability, of receiving their transplant at 1 year. The only factor associated with a lower probability of MSD SCT in first remission was delayed HLA typing (HR=0.82; P=0.03). One year after enrollment, 40% of patients did not follow their initial treatment plan. Because OS was 50% only at 3 years and only 57% of the patients without a MSD underwent SCT, this suggests room for improvement in outcomes for adults with acute leukemia.

摘要

对于成年急性白血病患者而言,了解最初计划的治疗方案是否真正得到实施至关重要。欧洲血液与骨髓移植学会急性白血病工作组对695例在进行HLA分型时登记的连续患者进行了前瞻性随访。在304例有可用的同胞全相合供者(MSD)的患者中,计划进行异基因造血干细胞移植(SCT)的有264例,计划进行化疗的有33例,计划进行自体造血干细胞移植的有7例。其余患者中,198例启动了无关供者(UD)搜索。其中,117例接受了移植,114例接受了化疗,77例接受了自体造血干细胞移植。在1年和2年时接受计划治疗的概率分别为60%和65%。计划接受MSD-SCT的患者在1年时接受移植的概率为82%,而计划接受UD-SCT的患者这一概率为57%。首次缓解期MSD-SCT概率较低的唯一相关因素是HLA分型延迟(风险比=0.82;P=0.03)。入组1年后,40%的患者未遵循其初始治疗计划。由于3年时总生存率仅为50%,且没有MSD的患者中只有57%接受了SCT,这表明成年急性白血病患者的治疗效果仍有改善空间。

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本文引用的文献

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Haplo-BMT: which approach?单倍体相合造血干细胞移植:哪种方法?
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Impact of donor-specific anti-HLA antibodies on graft failure and survival after reduced intensity conditioning-unrelated cord blood transplantation: a Eurocord, Société Francophone d'Histocompatibilité et d'Immunogénétique (SFHI) and Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) analysis.供者特异性抗 HLA 抗体对非亲缘性脐血移植后移植物失败和存活的影响:Eurocord、法国组织相容性和免疫遗传学学会(SFHI)和法国骨髓和细胞治疗移植学会(SFGM-TC)的分析。
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首次完全缓解期急性髓系白血病的异基因造血细胞移植:临床视角
Int J Hematol. 2015 Mar;101(3):243-54. doi: 10.1007/s12185-014-1657-0. Epub 2014 Sep 12.
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Should persons with acute myeloid leukemia have a transplant in first remission?急性髓系白血病患者应在首次缓解期进行移植吗?
Leukemia. 2014 Oct;28(10):1949-52. doi: 10.1038/leu.2014.129. Epub 2014 Apr 14.
Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia.
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Blood. 2013 Jan 31;121(5):752-8. doi: 10.1182/blood-2012-08-449108. Epub 2012 Dec 9.
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HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease.HLA 单倍体相合骨髓移植联合移植后环磷酰胺可扩大镰状细胞病患者的供者池。
Blood. 2012 Nov 22;120(22):4285-91. doi: 10.1182/blood-2012-07-438408. Epub 2012 Sep 6.
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The European LeukemiaNet AML Working Party consensus statement on allogeneic HSCT for patients with AML in remission: an integrated-risk adapted approach.欧洲白血病网络 AML 工作组关于缓解期 AML 患者异基因 HSCT 的共识声明:一种综合风险适应方法。
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The EBMT activity survey: 1990-2010.《EBMT 活动调查:1990-2010》。
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The superiority of haploidentical related stem cell transplantation over chemotherapy alone as postremission treatment for patients with intermediate- or high-risk acute myeloid leukemia in first complete remission.在首次完全缓解的中高危急性髓系白血病患者中,与单纯化疗相比,单倍体相合相关干细胞移植作为缓解后治疗具有优越性。
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Cord blood meets its match.脐血找到了匹配对象。
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Autologous peripheral blood stem cell transplantation for acute myeloid leukemia.自体外周血造血干细胞移植治疗急性髓细胞白血病。
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The EBMT activity survey 2009: trends over the past 5 years.2009 年 EBMT 活动调查:过去 5 年的趋势。
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