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首页> 外文期刊>The New England journal of medicine >A promising new treatment for refractory aplastic anemia
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A promising new treatment for refractory aplastic anemia

机译：难治性再生障碍性贫血的有希望的新疗法

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Aplastic anemia is characterized by severe deficiencies in peripheral-blood platelets, white cells, and red cells. These defects in mature cells occur because aplastic bone marrow contains severely reduced numbers of hematopoietic stem cells and their lineage-committed progenitor-cell progeny. In rare instances, the disease is familial, but in the majority of acquired cases, the origin of the disease is unclear, and the most favored hypothesis is that acquired aplastic anemia may be an immunologically mediated disease.The preferred treatment is immunotherapy with antithymocyte globulin plus cyclosporine. If this treatment proves ineffective, eligible patients can undergo allogeneic bone marrow transplantation. Although either treatment strategy can achieve long-term control of the disease, a considerable fraction of patients have disease that is refractory to these treatments.1'2 For such patients, an alternative approach is urgently needed.
机译：再生障碍性贫血的特征是外周血血小板，白细胞和红细胞严重缺乏。由于再生障碍性骨髓中含有大量减少的造血干细胞及其沿袭组成的祖细胞后代，因此会出现成熟细胞中的这些缺陷。在极少数情况下，该疾病为家族性疾病，但在大多数获得性病例中，疾病的起源尚不清楚，最受支持的假说是获得性再生障碍性贫血可能是免疫介导的疾病。首选治疗方法是用抗胸腺细胞球蛋白免疫疗法加环孢霉素。如果证明这种治疗无效，则符合条件的患者可以接受异体骨髓移植。尽管任何一种治疗策略都可以实现对该病的长期控制，但是相当一部分患者患有这些治疗难以治愈的疾病。1'2对于此类患者，迫切需要一种替代方法。

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