Department of Oncology, Sidney Kimmel Cancer Center at Johns Hopkins, Baltimore, MD, USA.
Department of Oncology, Sidney Kimmel Cancer Center at Johns Hopkins, Baltimore, MD, USA.
Best Pract Res Clin Haematol. 2021 Jun;34(2):101280. doi: 10.1016/j.beha.2021.101280. Epub 2021 Jun 26.
Hypoplastic MDS is a subset of MDS characterized by marrow hypocellularity diagnosed in 10-15% of MDS patients. The pathogenesis of this disease shares features of aplastic anemia with activation of the effector T cells against hematopoietic stem and progenitor cells and high-risk MDS with acquisition of somatic mutations that provide survival and growth advantage of these cells in the inflammatory bone marrow microenvironment. Clonal evolution in hypoplastic MDS may be associated with accumulation of DNA damage and progression to AML while clonal hematopoiesis in aplastic anemia is strongly related to immune escape of the hematopoietic cells. Distinction of hypoplastic MDS from other acquired and inherited bone marrow failure syndromes is frequently challenging but it is critical for the appropriate clinical management of the patients. Treatment with immunosuppression is an important component of the clinical approach to patients with hypoplastic MDS while hypomethylating agents and early allogeneic bone marrow transplantation are also considerations in some patients. In this review, we summarize the current literature on the biology of hypoplastic MDS, the differences between this disease and other bone marrow failure syndromes, and the treatment algorithm for patients with this subtype of MDS.
低增生性 MDS 是 MDS 的一个亚类，其特征为骨髓细胞减少，在 10-15%的 MDS 患者中诊断。这种疾病的发病机制与再生障碍性贫血具有共同特征，即效应 T 细胞对造血干细胞和祖细胞的激活，以及高危 MDS 中获得的体细胞突变，这些突变为这些细胞在炎症性骨髓微环境中提供了生存和生长优势。低增生性 MDS 中的克隆进化可能与 DNA 损伤的积累和向 AML 的进展有关，而再生障碍性贫血中的克隆性造血与造血细胞的免疫逃逸密切相关。将低增生性 MDS 与其他获得性和遗传性骨髓衰竭综合征区分开来常常具有挑战性，但对于患者的适当临床管理至关重要。免疫抑制治疗是低增生性 MDS 患者临床治疗方法的重要组成部分，而低甲基化剂和早期异基因骨髓移植也是某些患者的考虑因素。在这篇综述中，我们总结了低增生性 MDS 的生物学、该疾病与其他骨髓衰竭综合征的区别以及该亚型 MDS 患者的治疗方案的现有文献。
