PIEZO1 红细胞膜缺陷导致的遗传性溶血性贫血。

IF 1.2 4区 医学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY
Hemoglobin Pub Date : 2024-09-01 Epub Date: 2024-11-26 DOI:10.1080/03630269.2024.2427187
Georgios Dryllis, Roberta Russo, Immacolata Andolfo, Achille Iolascon, Barbara Eleni Rosato, Kostas Konstantopoulos
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引用次数: 0

摘要

PIEZO1(piezo-type mechanosensitive ion channel component 1)是一种机械敏感性离子通道蛋白。已知 PIEZO1 基因的功能增益变异可导致脱水型遗传性口腔细胞增多症(DHS),也称为遗传性脱水型口腔细胞增多症。这是一种罕见的常染色体显性遗传病,其特征是不同程度的贫血,有溶血、红细胞脱水和铁超载的倾向。虽然 DHS 的诊断流程已经确立,但由于其临床特征与其他溶血性贫血症重叠,且 PIEZO1 变体具有多效应,因此往往会延误诊断。我们描述了一例希腊患者的病例,该患者自出生起就患有代偿性溶血。从幼年到 70 岁,经过长时间的反复检查,最终确诊为 DHS。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Hereditary Hemolytic Anemia Due to PIEZO1 Red Blood Cell Membrane Defect.

PIEZO1 (piezo-type mechanosensitive ion channel component 1) is a mechanosensitive ion channel protein. Gain-of-function variants in the PIEZO1 gene are known to cause dehydrated hereditary stomatocytosis (DHS) also termed hereditary xerocytosis. This is a rare autosomal dominant condition characterized by variable-degree anemia with a tendency toward hemolysis, erythrocyte dehydration and iron overload. While the diagnostic workflow for DHS is well-established, diagnosis is often delayed due to overlapping clinical features with other hemolytic anemias and the pleiotropic effects of PIEZO1 variants. We describe the case of a Greek patient with a compensating hemolysis since birth. DHS diagnosis was established only after a prolonged history of repeated investigations spanning from his early life to 70 years of age, when a conclusive testing was achieved.

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来源期刊
Hemoglobin
Hemoglobin 医学-生化与分子生物学
CiteScore
1.70
自引率
10.00%
发文量
59
审稿时长
3 months
期刊介绍: Hemoglobin is a journal in the English language for the communication of research and information concerning hemoglobin in humans and other species. Hemoglobin publishes articles, reviews, points of view The journal covers topics such as: structure, function, genetics and evolution of hemoglobins biochemical and biophysical properties of hemoglobin molecules characterization of hemoglobin disorders (variants and thalassemias), consequences and treatment of hemoglobin disorders epidemiology and prevention of hemoglobin disorders (neo-natal and adult screening) modulating factors methodology used for diagnosis of hemoglobin disorders
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