Defective erythroid progenitor differentiation system in congenital hypoplastic (Diamond-Blackfan) anemia.
Defective erythroid progenitor differentiation system in congenital hypoplastic (Diamond-Blackfan) anemia.
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DOI：
10.1182/blood.v67.4.962.962
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发表时间：
1986-04
期刊：
影响因子：
20.3
通讯作者：
J. Lipton;M. Kudisch;R. Gross;D. Nathan
中科院分区：
文献类型：
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作者：
J. Lipton;M. Kudisch;R. Gross;D. Nathan
为了探讨先天性发育不良或钻石黑粉贫血(DBA)的病因，我们研究了9例患者的体外红细胞生成。在这9名患者中，有7名对强的松有临床反应。其中四个是在诊断时进行评估的婴儿。其中6人从未或仅接受过微量输血。那些服用了强的松的人在研究前至少五个月停止了药物治疗。将这9例患者的骨髓与血液学正常者及4例红系再生障碍性贫血的儿童一过性红细胞减少症患者进行比较。使用血浆凝块半固体培养技术计数红系祖细胞并评估其生长特性，我们得出结论：(A)红系祖细胞频率与贫血和红细胞减少的程度无关；(B)红系祖细胞分化在某些情况下可能对促红细胞生成素粗制剂异常不敏感；(C)体外促红细胞生成素不敏感并不一定表明体内对强的松不敏感。因此，DBA似乎不仅仅是红系祖细胞形成不足的结果，而且还是一种由于体内祖细胞分化缺陷所致的疾病。
To explore the etiology of congenital hypoplastic or Diamond-Blackfan anemia (DBA) we investigated in vitro erythropoiesis in nine patients. Of the nine, seven were clinically responsive to prednisone. Four were infants evaluated at the time of diagnosis. Six were never or were only minimally transfused. Those for whom prednisone had been prescribed had discontinued the drug a minimum of five months prior to study. The bone marrows of these nine patients were compared with those of hematologically normal individuals and with those of four patients with transient erythroblastopenia of childhood (TEC) whose erythroid aplasia was as severe as that of the patients with DBA. Using the plasma clot semisolid culture technique to enumerate erythroid progenitors and to evaluate the growth characteristics of the colonies to which they give rise, we concluded that at the onset of DBA: (a) erythroid progenitor frequency does not correlate with the degree of anemia and erythroblastopenia; (b) erythroid progenitor differentiation may in some cases be abnormally insensitive to crude preparations of erythropoietin; and (c) progenitor erythropoietin insensitivity in vitro does not necessarily indicate prednisone insensitivity in vivo. Thus, DBA does not appear to be solely the result of deficient formation of erythroid progenitors but is, in addition, a disorder that is due to defective progenitor differentiation in vivo.