University Children's Hospital, Philipps-University, Marburg, Germany.
INSERM/UPMC/CNRS-U1138, ERL8228, Team 3, Paris Cedex 06, France.
Curr Opin Nephrol Hypertens. 2018 Jul;27(4):323-328. doi: 10.1097/MNH.0000000000000422.
Antenatal Bartter's syndrome (aBS) is the most severe form of Bartter's syndrome, requiring close follow-up, in particular during the neonatal period, primarily because of prematurity. The recent identification of a novel and very severe form of aBS merits an update on this topic.
Despite the identification of several genes involved in Bartter's syndrome, about 20% of patients clinically diagnosed with aBS remained without genetic explanation for decades. We recently identified mutations in MAGED2 as a cause of an X-linked form of aBS characterized by a very early onset of severe polyhydramnios and extreme prematurity leading to high mortality. Remarkably, all symptoms in surviving patients with MAGE-D2 mutations resolve spontaneously, within weeks after preterm birth. Interestingly, MAGE-D2 affects the expression of the sodium chloride cotransporters NKCC2 and NCC, explaining thereby the severity of the disease. Importantly, a more recent analysis of MAGED2 in a large French cohort of patients with aBS confirmed our data and showed that females can also be affected.
MAGE-D2 is critical for renal salt reabsorption in the fetus, amniotic fluid volume regulation, and maintenance of pregnancy. Most importantly, MAGED2 must be included in the genetic screening of every form of aBS.
产前巴特综合征(aBS)是巴特综合征中最严重的一种,需要密切随访,特别是在新生儿期,主要是因为早产。最近发现了一种新型且非常严重的 aBS 形式,值得对此主题进行更新。
尽管已经确定了几个与巴特综合征相关的基因,但临床上被诊断为 aBS 的患者中有约 20%在几十年内仍无法用遗传解释。我们最近发现 MAGED2 的突变是一种 X 连锁形式的 aBS 的原因,其特征为严重羊水过多和早产,导致极高的死亡率。值得注意的是,所有患有 MAGE-D2 突变的幸存患者的症状在早产数周后都会自发缓解。有趣的是,MAGE-D2 会影响钠氯共转运蛋白 NKCC2 和 NCC 的表达,从而解释了疾病的严重程度。重要的是,最近对患有 aBS 的大量法国患者队列中的 MAGED2 进行的分析证实了我们的数据,并表明女性也可能受到影响。
MAGE-D2 对胎儿肾盐重吸收、羊水体积调节和妊娠维持至关重要。最重要的是,MAGED2 必须纳入所有形式 aBS 的基因筛查中。