Mohamed Razan, Azzah Ghadah, Piel Barbara
Paediatric, Queen Elizabeth Hospital King's Lynn NHS Trust, King's Lynn, UK
Paediatrics, Queen Elizabeth Hospital King's Lynn NHS Trust, Kings Lynn, UK.
BMJ Case Rep. 2025 Apr 15;18(4):e262786. doi: 10.1136/bcr-2024-262786.
Diamond-Blackfan anaemia (DBA) is a rare congenital disorder characterised by pure red cell aplasia and often associated with various congenital anomalies. This chronic condition has a heterogeneous genetic profile, typically inherited in an autosomal dominant manner, though rare cases of autosomal recessive inheritance exist. Most patients (90%) exhibit symptoms before 12 months of age, with the median age of presentation and diagnosis being approximately 2 months. This report presents a unique case of DBA identified at birth, resulting in a preterm delivery. The patient continues to be managed with frequent blood transfusions.
先天性纯红细胞再生障碍性贫血(DBA)是一种罕见的先天性疾病,其特征为单纯红细胞再生障碍,常伴有各种先天性异常。这种慢性病具有异质性的基因谱,通常以常染色体显性方式遗传,不过也存在常染色体隐性遗传的罕见病例。大多数患者(90%)在12个月龄前出现症状,发病和诊断的中位年龄约为2个月。本报告介绍了一例出生时即确诊的DBA罕见病例,该病例导致了早产。该患者目前仍需频繁输血治疗。