Heimpel H
Medizinische Universitätsklinik, Ulm, Deutschland.
Acta Haematol. 2000;103(1):11-5. doi: 10.1159/000040998.
In severe aplastic anemia, disease-dependent mortality was high before allogeneic bone marrow transplantation (BMT) and immunosuppressive therapies (IST) including antilymphocyte globulin became available. However, under supportive therapy alone, spontaneous remissions were observed in up to 20% of severe cases, reflecting the natural course of the disease. Therefore, in evaluating new forms of treatment, one has to keep in mind that remission is not necessarily response, and that final proof of utility of any new therapy still requires a randomized study design. Transition to leukemia or myelodysplasia was rarely observed if the initial diagnosis was accurate. The much higher incidence of leukemias in patients treated by IST, but not by BMT is probably due to the better life expectancy of patients at risk, rather than to a leukemogenic potential of IST itself. 'Outdated' therapeutic modalities, such as androgens or splenectomy, may still be justified as an adjuvant therapy in selected cases.
在严重再生障碍性贫血中，在异基因骨髓移植（BMT）和包括抗淋巴细胞球蛋白在内的免疫抑制疗法（IST）出现之前，疾病相关死亡率很高。然而，仅在支持性治疗下，高达20%的严重病例出现了自发缓解，这反映了疾病的自然病程。因此，在评估新的治疗形式时，必须记住缓解不一定等同于反应，任何新疗法效用的最终证明仍需要随机研究设计。如果初始诊断准确，很少观察到向白血病或骨髓增生异常综合征的转变。接受IST而非BMT治疗的患者白血病发病率高得多，这可能是由于高危患者的预期寿命更长，而非IST本身具有致白血病潜力。“过时”的治疗方式，如雄激素或脾切除术，在某些特定病例中作为辅助治疗仍可能是合理的。
