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一种与非免疫性水肿和胎儿丢失相关的 Gardos 通道病。

A Gardos channelopathy associated with nonimmune hydrops and fetal loss.

机构信息

CHU de Nantes, Service de Génétique Médicale, Nantes, France.

CHU de Nantes, UF 9321 de Fœtopathologie et Génétique, Nantes, France.

出版信息

Clin Genet. 2022 Dec;102(6):543-547. doi: 10.1111/cge.14217. Epub 2022 Sep 13.

Abstract

Dehydrated hereditary stomatocytosis (DHS) (MIM#194380) is a rare autosomal dominant disorder of red blood cell permeability, characterized by a partially or fully compensated nonimmune hemolytic anemia. PIEZO1 is the major gene involved with hundreds of families described, some of which present transient perinatal edema of varying severity. A smaller subset of individuals harbors pathogenic variants in KCNN4, sometimes referred as "Gardos channelopathy." Up to now, only six pathogenic variants in KCNN4 have been reported in 13 unrelated families. Unlike PIEZO1-DHS, neither perinatal edema nor fetal loss has ever been observed linked to KCNN4-DHS. We report the first fetal loss due to non-immune hydrops fetalis related to a pathogenic 28 bp deletion (NM_002250.2: c.1109_1119+17del) in KCNN4. This observation underlies the need for very close monitoring of pregnancies when one parent is affected by DHS regardless of genotype (PIEZO1 or KCNN4).

摘要

遗传性口炎性腹泻(DHS)(MIM#194380)是一种罕见的常染色体显性红细胞渗透性疾病,其特征为部分或完全代偿性非免疫性溶血性贫血。PIEZO1 是涉及数百个已描述家族的主要基因,其中一些家族表现为不同严重程度的短暂围产期水肿。一小部分个体携带有 KCNN4 的致病性变异,有时被称为“Gardos 通道病”。到目前为止,在 13 个无关家族中仅报道了 6 个 KCNN4 中的致病性变异。与 PIEZO1-DHS 不同,KCNN4-DHS 与围产期水肿或胎儿丢失无关。我们报道了首例与 KCNN4 相关的致病性 28bp 缺失(NM_002250.2:c.1109_1119+17del)导致的非免疫性胎儿水肿相关的胎儿丢失。这一观察结果表明,无论基因型(PIEZO1 或 KCNN4)如何,当父母一方受到 DHS 影响时,需要对妊娠进行非常密切的监测。

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