Hamammdi Ahmad, Sultan Tamimi Kareem, Qabaha Adam, Bsharat Omar, Ibraheem Kareem, Batran Ahmad
Faculty of Medicine Palestine Polytechnic University Hebron Palestine.
Palestinian Clinical Research Center Bethlehem Palestine.
Clin Case Rep. 2025 Sep 7;13(9):e70875. doi: 10.1002/ccr3.70875. eCollection 2025 Sep.
Congenital dyserythropoietic anemia type III (CDA III) is an extremely rare inherited disorder characterized by ineffective erythropoiesis, multinucleated erythroblasts in the bone marrow, and variable clinical gravity. We report the case of a 6-year-old boy, presenting with abdominal distension, failure to thrive, dark urine, intermittent itching, and recurrent infections. Physical examination revealed pallor, hepatomegaly, and splenomegaly. Laboratory investigations showed mild normocytic anemia, high liver enzymes, and erythroid hyperplasia. The sequencing of the whole exoma identified a variant of uncertain meaning in the KIF23 gene, which is implicated in cytokinesis and attached to the pathogenesis of CDA III. The patient remains clinically stable in support management, without the current need for transfusion. This case highlights the importance of advanced genetic tests in the diagnosis of rare hematological conditions and expands the potential spectrum of mutations associated with CDA III. Early recognition and long-term monitoring are essential for guiding management and monitoring complications such as iron overload and splenomegaly.
III型先天性红细胞生成异常性贫血(CDA III)是一种极为罕见的遗传性疾病,其特征为红细胞生成无效、骨髓中出现多核成红细胞以及临床严重程度各异。我们报告了一名6岁男孩的病例,该男孩表现为腹胀、发育不良、深色尿液、间歇性瘙痒和反复感染。体格检查发现面色苍白、肝肿大和脾肿大。实验室检查显示轻度正细胞性贫血、高肝酶和红系增生。全外显子组测序在KIF23基因中鉴定出一个意义未明的变异,该基因与胞质分裂有关,并与CDA III的发病机制相关。该患者在支持治疗下临床保持稳定,目前无需输血。该病例凸显了先进基因检测在罕见血液疾病诊断中的重要性,并扩展了与CDA III相关的潜在突变谱。早期识别和长期监测对于指导管理以及监测诸如铁过载和脾肿大等并发症至关重要。