Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations.
Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations.
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DOI：
10.1080/17474086.2016.1268048
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发表时间：
2017-01
影响因子：
2.8
通讯作者：
Wagner JE
中科院分区：
文献类型：
--
作者：
Ebens CL;MacMillan ML;Wagner JE
简介：在过去的40年里，范可尼贫血（FA）的造血细胞移植有了显着的改善。随着对造血衰竭和白血病发生的内在DNA修复缺陷和病理生理学的理解的增强，对预处理和移植物工程的连续改变显著改善了异基因造血细胞移植（alloHCT）后的存活预期，移植物衰竭的发生率从35%降低到<10%，急性移植物抗宿主病（GVHD）从>40%降低到<10%。今天，五年总生存率超过90%的年轻FA患者骨髓衰竭，但仍然约50%的血液系统malignances.Areas覆盖：我们审查的演变alloHCT有助于降低移植相关并发症的发生率;突出目前的挑战，包括不良后果的情况下，克隆性血液系统疾病，alloHCT的影响内分泌功能和固有的FA风险上皮恶性肿瘤;并描述了用于预防和治疗FA的血液学表现的研究性疗法。专家评论：目前的方法允许FA相关BMF在alloHCT后的极好存活，而与供体造血细胞来源无关。正在探索替代治疗方法，如基因治疗，以消除GVHD的风险，并尽量减少治疗相关的不良反应。
Introduction:Hematopoietic cell transplantation for Fanconi Anemia (FA) has improved dramatically over the past 40 years. With an enhanced understanding of the intrinsic DNA-repair defect and pathophysiology of hematopoietic failure and leukemogenesis, sequential changes to conditioning and graft engineering have significantly improved the expectation of survival after allogeneic hematopoietic cell transplantation (alloHCT) with incidence of graft failure decreased from 35% to <10% and acute graft-versus-host disease (GVHD) from >40% to <10%. Today, five-year overall survival exceeds 90% in younger FA patients with bone marrow failure but remains about 50% in those with hematologic malignancy.Areas covered:We review the evolution of alloHCT contributing to decreased rates of transplant related complications; highlight current challenges including poorer outcomes in cases of clonal hematologic disorders, alloHCT impact on endocrine function and intrinsic FA risk of epithelial malignancies; and describe investigational therapies for prevention and treatment of the hematologic manifestations of FA.Expert commentary:Current methods allow for excellent survival following alloHCT for FA associated BMF irrespective of donor hematopoietic cell source. Alternative curative approaches, such as gene therapy, are being explored to eliminate the risks of GVHD and minimize therapy-related adverse effects.
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影响因子：
20.3
作者：
BUTTURINI, A;GALE, RP;AUERBACH, AD
通讯作者：
AUERBACH, AD
影响因子：
4.3
作者：
Bonfim, Carmem;Ribeiro, Lisandro;Pasquini, Ricardo
通讯作者：
Pasquini, Ricardo
影响因子：
5.5
作者：
Alter, Blanche P.;Giri, Neelam;Pinto, Ligia A.
通讯作者：
Pinto, Ligia A.
影响因子：
4.8
作者：
Ayas, M.;Al-Jefri, A.;El-Solh, H.
通讯作者：
El-Solh, H.
影响因子：
45.3
作者：
Ayas, Mouhab;Saber, Wael;Gale, Robert Peter
通讯作者：
Gale, Robert Peter