Zhu Xiaoning, Peng Mengyun, Yin Yue, Zhang Yurong, Zheng Ding, Peng Zhaoxuan, Cheng Jun, Yang Song, Wang Jing
Department of Hepatobiliary Disease, The Affiliated Traditional Chinese Medicine Hospital of Southwest Medical University, Luzhou, Sichuan 646000, P.R. China.
Center of Liver Diseases, Beijing Ditan Hospital, Capital Medical University, Beijing 100020, P.R. China.
Exp Ther Med. 2022 Nov 16;25(1):4. doi: 10.3892/etm.2022.11704. eCollection 2023 Jan.
The present study describes the clinical profile and ankyrin 1 (ANK1) mutation status of a Chinese family with hereditary spherocytosis (HS). A young male patient (proband) was diagnosed with HS after presenting with anaemia and jaundice. The Coombs test was negative and spherocytes were found in peripheral blood smears. Magnetic resonance imaging showed splenomegaly and splenic iron depositions. The red blood cell osmotic fragility test was positive. The eosin-5'-maleimide binding test showed reduced mean channel fluorescence. Whole-exome sequencing revealed a novel ANK1 mutation (c.4707G>A), resulting in a nonsense mutation (p.Trp1569*). The patient's father, paternal aunt and paternal grandmother exhibited comparable clinical symptoms and Sanger sequencing confirmed the same mutation in these family members. To the best of our knowledge, an HS pedigree with this novel ANK1 nonsense mutation has not been previously reported. At the same time, the unique clinical presentation of this pedigree helps our understanding of the heterogeneity of clinical manifestations of HS.
本研究描述了一个患有遗传性球形红细胞增多症(HS)的中国家庭的临床特征和锚蛋白1(ANK1)突变状态。一名年轻男性患者(先证者)在出现贫血和黄疸后被诊断为HS。库姆斯试验呈阴性,外周血涂片发现球形红细胞。磁共振成像显示脾肿大和脾脏铁沉积。红细胞渗透脆性试验呈阳性。嗜酸性-5'-马来酰亚胺结合试验显示平均通道荧光降低。全外显子测序揭示了一种新的ANK1突变(c.4707G>A),导致无义突变(p.Trp1569*)。患者的父亲、姑姑和祖母表现出类似的临床症状,桑格测序证实这些家庭成员存在相同的突变。据我们所知,此前尚未报道过具有这种新的ANK1无义突变的HS家系。同时,该家系独特的临床表现有助于我们理解HS临床表现的异质性。