Speck B
Schweiz Med Wochenschr. 1986 Oct 25;116(43):1467-74.
In vitro cultures of hemopoietic precursor cells and clinical studies have partly clarified the pathogenesis of aplastic anemia. The defect is located both in the hemopoietic precursor cells and in their microenvironment. The therapeutic possibilities have enormously improved. Allogeneic bone marrow transplantation remains largely limited to young patients with an HLA-identical sibling donor. In our experience successfully transplanted patients are completely cured. Mortality from rejection, graft-versus-host-disease and infection has been markedly reduced. Immune modulating therapy with ALG produces high remission rates but residual defects in hemopoietic precursor cells remain and clonal disorders such as paroxysmal nocturnal hemoglobinuria may ensue. Despite of this ALG is the treatment of choice for patients without an HLA identical sibling donor.
造血前体细胞的体外培养及临床研究已部分阐明了再生障碍性贫血的发病机制。缺陷既存在于造血前体细胞，也存在于其微环境中。治疗的可能性有了极大改善。异基因骨髓移植在很大程度上仍仅限于有 HLA 相同同胞供者的年轻患者。根据我们的经验，成功接受移植的患者可完全治愈。排斥反应、移植物抗宿主病和感染导致的死亡率已显著降低。用抗淋巴细胞球蛋白进行免疫调节治疗可产生较高的缓解率，但造血前体细胞仍存在残余缺陷，可能会引发诸如阵发性夜间血红蛋白尿等克隆性疾病。尽管如此，抗淋巴细胞球蛋白仍是没有 HLA 相同同胞供者的患者的首选治疗方法。
