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骨髓增生异常综合征中的免疫抑制治疗:一种寻找合适应用场景的舶来疗法。

Immunosuppressive therapy in myelodysplastic syndromes: a borrowed therapy in search of the right place.

作者信息

Shallis Rory M, Chokr Nora, Stahl Maximilian, Pine Alexander B, Zeidan Amer M

机构信息

a Division of Hematology/Medical Oncology, Department of Medicine , Yale University School of Medicine , New Haven , USA.

b Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center , Yale University , New Haven , USA.

出版信息

Expert Rev Hematol. 2018 Sep;11(9):715-726. doi: 10.1080/17474086.2018.1503049. Epub 2018 Aug 22.

Abstract

Myelodysplastic syndromes (MDS) encompass a heterogenous collection of clonal hematopoietic stem cell disorders defined by dysregulated hematopoiesis, peripheral cytopenias, and a risk of leukemic progression. Increasing data support the role of innate and adaptive immune pathways in the pathogenesis and disease course of MDS. The role of immunosuppressive therapy has an established role in the treatment of other hematologic diseases, such as aplastic anemia whose pathogenesis is postulated to reflect that of MDS with regards to many aspects of immune activation. Areas covered: This paper discusses the current understanding of immune dysregulation as it pertains to MDS, the clinical experience with immunosuppressive therapy in the management of MDS, as well as future prospects which will likely improve therapeutic options and outcomes for patients with MDS. Expert commentary: Though limited by paucity of high quality data, immunomodulatory and immunosuppressive therapies for the treatment of MDS have shown meaningful clinical activity in selected patients. Continued clarification of the immune pathways that are dysregulated in MDS and establishing predictors for clinical benefit of immunosuppressive therapy are vital to improve the use and outcomes with these therapies.

摘要

骨髓增生异常综合征(MDS)是一组异质性的克隆性造血干细胞疾病,其特征为造血功能失调、外周血细胞减少以及白血病进展风险。越来越多的数据支持先天性和适应性免疫途径在MDS发病机制和病程中的作用。免疫抑制疗法在其他血液系统疾病的治疗中已确立了作用,例如再生障碍性贫血,其发病机制在免疫激活的许多方面被认为与MDS相似。涵盖领域:本文讨论了目前对与MDS相关的免疫失调的理解、免疫抑制疗法在MDS管理中的临床经验,以及未来可能改善MDS患者治疗选择和预后的前景。专家评论:尽管受到高质量数据匮乏的限制,但用于治疗MDS的免疫调节和免疫抑制疗法在部分患者中已显示出有意义的临床活性。持续阐明MDS中失调的免疫途径并建立免疫抑制疗法临床获益的预测指标,对于改善这些疗法的应用和疗效至关重要。

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