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阐明慢性骨髓增生性恶性肿瘤的新生物学方面和潜在的新治疗方法。

Illuminating novel biological aspects and potential new therapeutic approaches for chronic myeloproliferative malignancies.

机构信息

Tufts University Medical Center, Boston, Massachusetts, USA.

MD Anderson Cancer Center, Houston, Texas, USA.

出版信息

Hematol Oncol. 2020 Dec;38(5):654-664. doi: 10.1002/hon.2771. Epub 2020 Sep 4.

Abstract

This review reflects the presentations and discussion at the 14th post-American Society of Hematology (ASH) International Workshop on Chronic Myeloproliferative Malignancies, which took place on the December 10 and 11, 2019, immediately after the 61st ASH Annual Meeting in Orlando, Florida. Rather than present a resume of the proceedings, we address some of the topical translational science research and clinically relevant topics in detail. We consider how recent studies using single-cell genomics and other molecular methods reveal novel aspects of hematopoiesis which in turn raise the possibility of new therapeutic approaches for patients with myeloproliferative neoplasms (MPNs). We discuss how alternative therapies could benefit patients with chronic myeloid leukemia who develop BCR-ABL1 mutant subclones following ABL1-tyrosine kinase inhibitor therapy. In MPNs, we focus on efforts beyond JAK-STAT and the merits of integrating activin receptor ligand traps, interferon-α, and allografting in the current treatment algorithm for patients with myelofibrosis.

摘要

这篇综述反映了在美国血液学会(ASH)第 14 次国际慢性骨髓增殖性恶性肿瘤研讨会上的演讲和讨论,该研讨会于 2019 年 12 月 10 日和 11 日在佛罗里达州奥兰多举行,紧随第 61 届 ASH 年会之后。我们没有对会议进程进行总结,而是详细探讨了一些热门转化科学研究和临床相关话题。我们考虑了最近使用单细胞基因组学和其他分子方法的研究如何揭示造血的新方面,这反过来又为骨髓增殖性肿瘤(MPN)患者提供了新的治疗方法的可能性。我们讨论了替代疗法如何使慢性髓性白血病患者受益,这些患者在接受 ABL1-酪氨酸激酶抑制剂治疗后会出现 BCR-ABL1 突变亚克隆。在 MPN 中,我们关注超出 JAK-STAT 的努力,以及整合激活素受体配体陷阱、干扰素-α 和同种异体移植在骨髓纤维化患者当前治疗方案中的优点。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6b5d/8895354/70016730bdbc/nihms-1783486-f0001.jpg

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