Camitta B M, Doney K
Department of Pediatrics, Medical College of Wisconsin, Milwaukee.
Am J Pediatr Hematol Oncol. 1990 Winter;12(4):411-24. doi: 10.1097/00043426-199024000-00005.
Autoimmune phenomena may be detected in patients with aplastic anemia. In the etiology of aplastic anemia, it is not known whether these processes are primary factors (causative), perpetuating factors (following other causative factors), or merely epiphenomena. Response to immunosuppressive therapy would suggest a causative or perpetuating role for autoimmunity in development of this disease. We have reviewed trials of immunosuppressive therapy, including the use of antilymphocyte globulin, cyclophosphamide, high-dose corticosteroids, cyclosporine, and apheresis, as well as combinations of these agents, for treatment of aplastic anemia. Responses occur in 40-70% of patients who receive various preparations of antilymphocyte globulin. Responses to other immunosuppressive regimens have been less frequent. Responses to all immunosuppressive regimens are usually incomplete, and late complications, such as relapse, paroxymal noctural hemoglobinuria, or leukemia, are being reported with increasing frequency. For all treatments, potential mechanisms of action other than immune suppression are possible. HLA-matched sibling-donor bone marrow transplantation is the treatment of choice for children with severe aplastic anemia. For children without a matched sibling, antilymphocyte globulin is the best current therapy. No treatment has been shown to alter the long-term course of mild aplastic anemia. Other immunosuppressive agents and transplant regimens should be considered experimental. The therapeutic value of these modalities can only be established by well-monitored trials performed at centers with special clinical and laboratory expertise.
再生障碍性贫血患者可能会检测到自身免疫现象。在再生障碍性贫血的病因中，尚不清楚这些过程是主要因素（致病因素）、持续因素（继其他致病因素之后），还是仅仅是附带现象。对免疫抑制治疗的反应提示自身免疫在该疾病发展中具有致病或持续作用。我们回顾了免疫抑制治疗的试验，包括使用抗淋巴细胞球蛋白、环磷酰胺、大剂量皮质类固醇、环孢素和血液成分单采术，以及这些药物的联合使用，用于治疗再生障碍性贫血。接受各种抗淋巴细胞球蛋白制剂治疗的患者中，40% - 70%会出现反应。对其他免疫抑制方案的反应则较少见。对所有免疫抑制方案的反应通常不完全，并且诸如复发、阵发性夜间血红蛋白尿或白血病等晚期并发症的报道频率正在增加。对于所有治疗方法，除免疫抑制外的潜在作用机制也是可能的。人类白细胞抗原（HLA）匹配的同胞供体骨髓移植是重症再生障碍性贫血儿童的首选治疗方法。对于没有匹配同胞的儿童，抗淋巴细胞球蛋白是目前最佳的治疗方法。尚无治疗方法被证明能改变轻度再生障碍性贫血的长期病程。其他免疫抑制剂和移植方案应被视为实验性的。这些治疗方式的治疗价值只能通过在具有特殊临床和实验室专业知识的中心进行的严密监测试验来确定。
