Treatment of high-risk myelodysplastic syndromes.

IF 8.2 1区 医学 Q1 HEMATOLOGY
Nicolaus Kröger
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Abstract

Myelodysplastic syndrome (MDS) is considered to be a heterogeneous myeloid malignancy with a common origin in the hematopoietic stem cell compartment and is generally divided into lower- and higher-risk forms. While the treatment goals for lower-risk MDS are to decrease transfusion requirements and transformation into acute leukemia, the major aims for higher-risk MDS are to prolong survival and ultimately cure the patient. Although novel agents such as luspatercept and imetelstat have recently been approved as new treatment options for lower-risk MDS, hypomethylating agents currently remain the only approved non-transplant option for higher-risk MDS and are the standard of care for patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). Combinations with other drugs as first-line treatment have to date not proven more efficacious than monotherapy in higher-risk MDS, and outcome after the failure of treatment with hypomethylating agents is poor. The only potential cure and standard of care for eligible patients is HSCT and even though the number of transplanted - especially older - MDS patients has increased over time due to better management and greater donor availability, the majority of MDS patients will not be eligible for this curative approach. Current challenges include decreasing the relapse risk, the main cause of HSCT failure. This review summarizes current knowledge on the options of transplant and non-transplant treatment approaches for these patients and demonstrate the unmet clinical need for more effective therapies.

高危骨髓增生异常综合征的治疗。
骨髓增生异常综合征(MDS)被认为是一种异质髓系恶性肿瘤,其共同起源为造血干细胞室,一般分为低危和高危。低风险MDS (LR-MDS)的治疗目标是减少输血负担和转变为急性白血病,而高风险MDS的治疗目标是延长生存期并最终治愈。虽然新型药物如luspatercept或imetelstat最近已被批准为LR-MDS的新治疗方案,但低甲基化药物(HMA)仍然是目前唯一批准的HR-MDS非移植治疗方案,也是非移植患者的标准治疗方案。迄今为止,在HR-MDS中,联合其他药物作为一线治疗尚未被证明比单药治疗更有效,HMA失败后的预后很差。对于符合条件的患者,唯一潜在的治疗方法和标准护理是异体干细胞移植(HSCT),即使移植的数量随着时间的推移而增加,特别是老年MDS患者,由于更好的管理和供体可用性,大多数MDS患者将不符合这种治疗方法。目前的挑战包括降低复发风险,这是HSCT失败的主要原因。这篇综述将总结目前对这些患者的移植和非移植治疗方法的选择,并展示对更有效治疗方法的未满足的临床需求。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Haematologica
Haematologica 医学-血液学
CiteScore
14.10
自引率
2.00%
发文量
349
审稿时长
3-6 weeks
期刊介绍: Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research. Scope: The scope of the journal includes reporting novel research results that: Have a significant impact on understanding normal hematology or the development of hematological diseases. Are likely to bring important changes to the diagnosis or treatment of hematological diseases.
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