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低危骨髓增生异常综合征患者的治疗方法:现有选择和未来方向。

Therapeutic modalities for patients with lower-risk myelodysplastic syndromes: current options and future directions.

机构信息

Tisch Cancer Institute, Mount Sinai School of Medicine, One Gustave L. Levy Place, Box 1079, New York, NY 10029, USA.

出版信息

Curr Hematol Malig Rep. 2011 Mar;6(1):5-12. doi: 10.1007/s11899-010-0071-9.

DOI:10.1007/s11899-010-0071-9
PMID:21153773
Abstract

The treatment of myelodysplastic syndromes (MDS) involves a complex algorithm that depends on multiple factors, including symptoms, performance status, and severity of disease. Current therapies are aimed at promoting hematopoiesis, inhibiting apoptosis, and reducing the risk of transformation to acute myeloid leukemia. Although there is no cure for MDS outside of allogeneic stem cell transplantation, goals of treatment include improvement of peripheral blood cytopenias, reduction of transfusions, improvement of the quality of life, and prolongation of survival. Patients with lower-risk MDS are often asymptomatic and can be monitored for long periods without therapeutic intervention. Anemia, the most common symptomatic cytopenia, warrants treatment in an attempt to eliminate transfusion dependence. This article reviews current treatment strategies for lower-risk MDS and examines the data for selected novel agents that are available or are being developed for the treatment of this disease.

摘要

骨髓增生异常综合征(MDS)的治疗涉及一个复杂的算法,该算法取决于多个因素,包括症状、体能状态和疾病严重程度。目前的治疗方法旨在促进造血、抑制细胞凋亡和降低转化为急性髓系白血病的风险。尽管除了异基因干细胞移植之外,MDS 没有其他治愈方法,但治疗的目标包括改善外周血细胞减少症、减少输血、改善生活质量和延长生存时间。低危 MDS 患者通常无症状,可以在没有治疗干预的情况下进行长期监测。贫血是最常见的有症状的细胞减少症,需要进行治疗,以试图消除对输血的依赖。本文综述了低危 MDS 的当前治疗策略,并研究了一些新型药物的数据,这些药物可用于或正在开发用于治疗这种疾病。

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本文引用的文献

1
Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions.阿仑单抗治疗中危-1 骨髓增生异常综合征患者可使血细胞计数和细胞遗传学缓解持续改善。
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Phase 2 study of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy.接受阿扎胞苷治疗的低危或中危骨髓增生异常综合征患者罗米司亭的 2 期研究。
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Incidence and clinical complications of myelodysplastic syndromes among United States Medicare beneficiaries.
美国医疗保险受益人群中骨髓增生异常综合征的发病率及临床并发症。
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Darbepoetin alfa in anemia of myelodysplastic syndromes: present and beyond.达贝泊汀α治疗骨髓增生异常综合征相关贫血:现状与展望。
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Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and thrombocytopenia.罗米司亭治疗低危骨髓增生异常综合征伴血小板减少症患者的安全性和有效性。
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Am J Hematol. 2009 Oct;84(10):671-7. doi: 10.1002/ajh.21503.
7
Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996).采用促红细胞生成素联合或不联合粒细胞集落刺激因子治疗骨髓增生异常综合征患者:东部肿瘤协作组(E1996)一项前瞻性随机3期试验的结果
Blood. 2009 Sep 17;114(12):2393-400. doi: 10.1182/blood-2009-03-211797. Epub 2009 Jun 29.
8
Phase 1-2a multicenter dose-escalation study of ezatiostat hydrochloride liposomes for injection (Telintra, TLK199), a novel glutathione analog prodrug in patients with myelodysplastic syndrome.盐酸依泽替米星脂质体注射液(TLK199,Ezatiostat)治疗骨髓增生异常综合征的 1/2a 期多中心剂量递增研究。TLK199 是一种新型谷胱甘肽类似物前药。
J Hematol Oncol. 2009 May 13;2:20. doi: 10.1186/1756-8722-2-20.
9
Phase 1 multicenter dose-escalation study of ezatiostat hydrochloride (TLK199 tablets), a novel glutathione analog prodrug, in patients with myelodysplastic syndrome.一项关于新型谷胱甘肽类似物前药盐酸依扎托司他(TLK199片)用于骨髓增生异常综合征患者的1期多中心剂量递增研究。
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Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.