Tooze J A, Marsh J C, Gordon-Smith E C
Department of Haematology, St George's Hospital Medical School, London, UK.
Leuk Lymphoma. 1999 Apr;33(3-4):231-41. doi: 10.3109/10428199909058423.
Aplastic anaemia (AA) is a non-malignant haemopoietic disorder characterised by peripheral blood pancytopenia and a hypocellular bone marrow. Successful management of acquired AA including treatment with immunosuppressive agents, mainly antithymocyte globulin (ATG) and cyclosporin or allogeneic haemopoietic stem cell transplantation, has resulted in long-term survival of many patients. The later evolution of complicating clonal disorders such as paroxysmal nocturnal haemoglobinuria, myelodysplasia and acute myeloid leukaemia in patients treated with immunosuppressive therapy may be a manifestation of the natural history of the aplasia, the development of which may or may not be increased by immunosuppressive therapy. A persistent, profound deficiency and/or defect in the stem cell compartment, despite haematological recovery after immunosuppressive therapy, may create an unstable situation which predisposes to later clonal disorders. A review of the progression of AA to clonal disorders is now outlined.
再生障碍性贫血（AA）是一种非恶性造血系统疾病，其特征为外周血全血细胞减少和骨髓细胞减少。获得性AA的成功治疗，包括使用免疫抑制剂（主要是抗胸腺细胞球蛋白（ATG）和环孢素）或异基因造血干细胞移植进行治疗，已使许多患者长期存活。接受免疫抑制治疗的患者中，诸如阵发性夜间血红蛋白尿、骨髓增生异常综合征和急性髓系白血病等并发克隆性疾病的后期演变，可能是再生障碍性贫血自然病程的一种表现，免疫抑制治疗可能会增加或不增加其发生。尽管免疫抑制治疗后血液学恢复，但干细胞池持续、严重的缺陷和/或缺陷可能会造成不稳定的情况，易引发后期的克隆性疾病。现将AA向克隆性疾病进展的综述概述如下。
