Hematology Branch, National Heart, Lung, and Blood Institute, and Center for Human Immunology, Autoimmunity, and Inflammation, National Institutes of Health, Bldg 10 CRC 3-1341, Bethesda, MD 20892, USA.
JAMA. 2011 Feb 23;305(8):814-9. doi: 10.1001/jama.2011.194.
Myelodysplastic syndromes (MDS) are a heterogeneous group of bone marrow disorders characterized by ineffective hematopoiesis and a tendency to develop leukemia. In some patients, laboratory and clinical evidence supports a role for the immune system in the pathogenesis of early MDS. Many younger patients who respond to immunosuppressive therapy with drugs such as antithymocyte globulin and cyclosporine have clonal expansions of cytotoxic CD8(+) T cells that suppress normal hematopoiesis, as well as expansion of CD4(+) helper T-cell subsets that promote and sustain autoimmunity. Immunosuppressive therapy can produce hematologic responses in some patients and may improve survival and halt leukemic progression. In this report, we describe a 56-year-old woman who presented with fatigue and easy bruising, eventually became pancytopenic, and was diagnosed with MDS. After treatment with a clinical protocol using alemtuzumab, an anti-CD52 antibody, her blood cell counts returned to normal and she has remained in complete remission for more than 2 years of follow-up. In this article, we review the pathobiology of immune dysregulation in MDS and summarize the role of immunosuppressive therapy in MDS.
骨髓增生异常综合征（MDS）是一组异质性的骨髓疾病，其特征为无效造血和发展为白血病的倾向。在一些患者中，实验室和临床证据支持免疫系统在早期 MDS 的发病机制中起作用。许多对免疫抑制治疗有反应的年轻患者，如抗胸腺细胞球蛋白和环孢素等药物，其细胞毒性 CD8(+) T 细胞会发生克隆扩张，从而抑制正常造血，同时也会促进和维持自身免疫的 CD4(+)辅助性 T 细胞亚群的扩张。免疫抑制治疗可在一些患者中产生血液学反应，并可能改善生存和阻止白血病进展。在本报告中，我们描述了一位 56 岁女性，她因疲劳和容易瘀伤就诊，最终发展为全血细胞减少，并被诊断为 MDS。在接受使用 alemtuzumab（一种抗 CD52 抗体）的临床方案治疗后，她的血细胞计数恢复正常，在 2 年多的随访中她一直处于完全缓解状态。在本文中，我们综述了 MDS 中免疫失调的病理生物学，并总结了免疫抑制治疗在 MDS 中的作用。
