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异基因造血细胞移植治疗未缓解 AML 患者:潜在治愈性疗法。

Allogeneic hematopoietic cell transplantation in patients with AML not achieving remission: potentially curative therapy.

机构信息

Memorial Sloan Kettering Cancer Center, New York, NY, USA.

Weill Cornell Medical College of Cornell University, New York, NY, USA.

出版信息

Bone Marrow Transplant. 2017 Aug;52(8):1083-1090. doi: 10.1038/bmt.2017.8. Epub 2017 Feb 27.

DOI:10.1038/bmt.2017.8
PMID:28244979
Abstract

Patients with acute myeloid leukemia (AML) who fail to achieve complete remission (CR) have a dismal prognosis. Although data suggest that durable remissions can be achieved in approximately 30% of patients with refractory or relapsed AML after allogeneic hematopoietic cell transplantation (HCT), only a small fraction of those patients are offered this therapeutic option. Importantly, patients with primary refractory AML have distinctly better outcomes following allogeneic HCT than those with refractory relapse. Access to suitable donors could be one of the main barriers in these situations. However, with recent developments in the field of allogeneic HCT, such as alternative donor sources, high-resolution HLA-typing, reduced intensity conditioning regimens and improvements in supportive care, this approach has the potential to offer long-term survival for patients with refractory and relapsed AML and should be considered as early after diagnosis as possible. Incorporating novel agents into the conditioning regimen or as post-transplant maintenance therapy could further improve outcomes and render older or medically infirm patients with refractory or relapsed AML eligible for allogeneic HCT. In this review, we summarize existing data on allogeneic HCT in patients with refractory or relapsed AML and explore novel approaches with the potential to improve outcomes in this patient population.

摘要

对于未能达到完全缓解 (CR) 的急性髓系白血病 (AML) 患者,预后较差。尽管数据表明,对于接受异基因造血细胞移植 (HCT) 的难治性或复发 AML 患者,约 30%的患者可以获得持久缓解,但只有一小部分患者有机会选择这种治疗方法。重要的是,原发性难治性 AML 患者在接受异基因 HCT 后的结局明显优于复发难治性 AML 患者。合适供体的获得可能是这些情况下的主要障碍之一。然而,随着异基因 HCT 领域的最新进展,如替代供体来源、高分辨率 HLA 分型、强度降低的预处理方案以及支持性护理的改善,这种方法有可能为难治性和复发性 AML 患者提供长期生存,并应在诊断后尽早考虑。将新型药物纳入预处理方案或作为移植后维持治疗可能进一步改善结局,并使年龄较大或身体虚弱的难治性或复发性 AML 患者有资格接受异基因 HCT。在这篇综述中,我们总结了难治性或复发性 AML 患者接受异基因 HCT 的现有数据,并探讨了具有改善该患者人群结局潜力的新方法。

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Haematologica. 2017 Jan;102(1):184-191. doi: 10.3324/haematol.2016.150326. Epub 2016 Aug 25.
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An operational definition of primary refractory acute myeloid leukemia allowing early identification of patients who may benefit from allogeneic stem cell transplantation.原发性难治性急性髓系白血病的操作性定义,有助于早期识别可能从异基因干细胞移植中获益的患者。
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改良马法兰和白消安方案联合维持治疗可改善异基因移植后难治性/复发性急性髓系白血病患者的生存率:一项多中心试验的中期结果
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