Residual hemopoiesis and myelodysplasia development in aplastic anemia children. -Epidemiologic and clinical observation

虽然再生障碍性贫血(AA)和骨髓增生异常综合征(MDS)-难治性贫血(RA)都是骨髓衰竭综合征，但这些疾病之间的疾病重叠已被讨论。本研究的目的是阐明MDS继发AA患者的病因和临床特征以及这些骨髓衰竭综合征中残留的造血作用。根据日本儿童血液学学会建立的数据库，MDS继发AA患者的预后取决于染色体异常。8三体是良好的预后指标，而7号单体是不良的预后标志。RA患者发生MDS的发生率较低。免疫抑制治疗(IST)的临床和血液学反应在AA和MDS-RA中都被怀疑高达50-60%。在两种类型的骨髓衰竭中，MCV也很高，因为MCV反映了无效的红细胞生成。为了评估残余造血，在一项小规模研究中引入了多维流式细胞术分析(MDF)。初步的结果是，MDF技术有可能在AA诊断时检测未成熟髓系细胞的耗竭，并能够跟踪IST后的造血恢复，也可以检测异常成熟途径上的异常Fells。

Although both of an aplastic anemia (AA) and a myelodysplastic syndrome(MDS)-refractory anemia(RA) are bone marrow failure syndrome, disease-overlapping between these disease has been discussed. Aim of this study is clarified that etiology and clinical characteristics of MDS-developed AA patient and residual hemopoiesis in these bone marrow failure syndrome.According to database that has been established by Japanese Society of Pediatric Hematology, a prognosis of MDS developed AA patient depends on chromosome abnormalities. Trisomy 8 is good, however monosomy 7 is poor prognostic marker. Incidence of MDS development in RA patients was low. Clinical and hematological response for immunosuppressive therapy (IST) was suspected as high as 50-60% in both AA and MDS-RA. MCV was also high in both type of bone marrow failure because MCV reflects ineffective erythropoiesis.For the purpose of evaluation for residual hemopoiesis, multidimensional flow cytometry analysis (MDF) was introduced in a small-scale study. As a tentative result, MDF technique has a possibility to detect exhaustion of immature myeloid cells at an AA diagnosis and was able to follow hemopoiesis recovery after IST, and also detect aberrant Fells on abnormal maturation pathway.