Decision Analysis of Transplantation for Patients with Myelodysplasia: "Who Should We Transplant Today?".

Decision Analysis of Transplantation for Patients with Myelodysplasia: "Who Should We Transplant Today?".
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DOI:
10.1007/s11899-020-00573-6
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发表时间:
2020-08
影响因子:
2.9
通讯作者:
Nakamura R
Nakamura R
中科院分区:
医学3区
文献类型:
--
作者:
Arslan S;Nakamura R

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骨髓增生异常综合征(MDS)是一种异质性血液学疾病,其特征是一系列临床表现、细胞遗传学和体细胞基因突变以及转化为急性白血病的风险。治疗方案包括观察、支持治疗、输血、使用生长因子和/或低甲基化药物以及在疾病进展前期或之后进行造血细胞移植(HCT)。目前,HCT是MDS患者唯一可用的治愈性治疗方法,在决定是否进行移植时涉及多种因素,如供体可用性、患者和疾病特征。在本文中,我们总结了1)MDS的总体预后和自然史,2)目前可用的非HCT治疗,重点是低甲基化药物(HMA), 3) MDS患者HCT后的结果,4)进行HCT治疗MDS需要考虑的因素,以及5)最近/正在进行的与HCT决策过程相关的研究。
Myelodysplastic syndrome (MDS) is a heterogeneous hematological disorder characterized by a spectrum of clinical presentation, cytogenetic, and somatic gene mutations and the risk of transformation to acute leukemia. Management options include observation, supportive care, blood transfusion, administration of growth factors and/or hypomethylating agents and hematopoietic cell transplant (HCT) either upfront or after disease progression. Currently, HCT is the only curative therapy available for patients with MDS, with multiple factors such as donor availability, patient and disease characteristics being involved in making the decision to proceed with transplant. In this article, we summarize 1) overall prognosis and natural history of MDS, 2) currently available non-HCT therapy with a focus on hypomethylating agents (HMA), 3) outcomes after HCT in patients with MDS, 4) factors to be considered to proceed to HCT for treatment of MDS, and 5) more recent/ongoing studies relevant to HCT decision making processes.
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