Glader B E
Department of Pediatrics, Stanford University School of Medicine, California.
Hematol Oncol Clin North Am. 1987 Sep;1(3):431-47.
The vast majority of pediatric RBC hypoplastic anemias are accounted for by red blood cell aplasia associated with chronic hemolysis, Diamond-Blackfan anemia, and transient erythroblastopenia of childhood. However, other causes of hypoplastic anemia occur in children, and some of these are similar to what is seen in adult RBC aplasia. For example, it has been reported that a 5-year-old girl with an aregenerative anemia had a thymoma and later developed pancytopenia. RBC aplasia also has been seen in children receiving anticonvulsant drug therapy, children recovering from severe protein malnutrition, children with hepatitis, and in children with leukemia during maintenance therapy. In addition, it is not uncommon for pediatric hematologists to observe children with RBC aplasia where there is no obvious diagnosis, although many are considered to be variants of Diamond-Blackfan anemia. Several important questions about RBC hypoplastic anemias in children need to be resolved; it is hoped that this will be accomplished in the next decade. Do RBC hypoplastic crises associated with hemolytic anemia occur with viral infections other than HPV? What is the cellular pathophysiology in DBA and TEC? Does the apparent heterogeneity of these disorders reflect limitations of laboratory techniques or are we looking at several different diseases? Is acute leukemia a real complication of Diamond-Blackfan anemia? Is TEC a completely benign entity or will we see other long-term problems in these children? Is the incidence of TEC actually increasing? Will TEC-like problems be seen in other aged children? As a case in point, we recently observed a 16-year-old girl who presented with pure RBC aplasia that required RBC transfusion support for 5 months; she also received prednisone therapy. After 7 months, however, this young lady had a spontaneous remission, and now 4 years later she is normal and free of any hematologic abnormalities. This was a most unusual event in our experience and, in view of the apparent increasing incidence of TEC in young children, we queried whether we were observing an adolescent equivalent of this disorder. During the next several years the answer to this and the other questions posed herein should be available.
绝大多数小儿红细胞发育不全性贫血是由与慢性溶血相关的红细胞再生障碍、先天性纯红细胞再生障碍性贫血（Diamond-Blackfan贫血）和儿童期短暂性红细胞生成减少症引起的。然而，小儿发育不全性贫血还有其他病因，其中一些与成人红细胞再生障碍所见相似。例如，有报道称一名5岁再生障碍性贫血女童患有胸腺瘤，随后发展为全血细胞减少症。在接受抗惊厥药物治疗的儿童、从严重蛋白质营养不良中恢复的儿童、患有肝炎的儿童以及白血病维持治疗期间的儿童中也可见到红细胞再生障碍。此外，小儿血液科医生观察到许多无明显诊断的红细胞再生障碍患儿并不罕见，尽管其中许多被认为是先天性纯红细胞再生障碍性贫血的变异型。关于小儿红细胞发育不全性贫血的几个重要问题有待解决；希望在未来十年内能够实现。与溶血性贫血相关的红细胞发育不全性危象是否会在除人乳头瘤病毒（HPV）以外的病毒感染时发生？先天性纯红细胞再生障碍性贫血和儿童期短暂性红细胞生成减少症的细胞病理生理学是什么？这些疾病明显的异质性是反映了实验室技术的局限性，还是我们正在研究几种不同的疾病？急性白血病是先天性纯红细胞再生障碍性贫血的真正并发症吗？儿童期短暂性红细胞生成减少症是完全良性的疾病，还是我们会在这些儿童中看到其他长期问题？儿童期短暂性红细胞生成减少症的发病率实际上在增加吗？在其他年龄段的儿童中会出现类似儿童期短暂性红细胞生成减少症的问题吗？举个例子，我们最近观察到一名16岁女孩，她表现为单纯红细胞再生障碍，需要红细胞输血支持5个月；她还接受了泼尼松治疗。然而，7个月后，这位年轻女士自发缓解，现在4年过去了，她一切正常，没有任何血液学异常。在我们的经验中，这是一个非常不寻常的事件，鉴于幼儿中儿童期短暂性红细胞生成减少症的发病率明显增加，我们质疑我们是否正在观察到这种疾病的青少年等效病例。在接下来的几年里，本文提出的这个问题和其他问题应该会有答案。
