SLC10A2变异的纯合子性与婴儿因胆汁酸吸收不良而发生严重脂溶性维生素缺乏症。

JPGN reports Pub Date : 2025-05-22 eCollection Date: 2025-08-01 DOI:10.1002/jpr3.70030
Christine Rungoe, Stefan Stender, Nawar Dalila, Emil D Bartels, Christian Jakobsen
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引用次数: 0

摘要

我们提出了一个病例的年轻女性患者持续和严重的脂溶性维生素缺乏症,因为婴儿期。尽管在儿童时期进行了广泛的调查,但根本原因仍然难以捉摸。患者在持续接受维生素补贴时,一般无症状。18岁时进行的外显子组测序显示,SLC10A2基因存在纯合错义变异(Pro65Leu),该基因编码回肠中的胆囊酸转运体。胆汁潴留扫描显示严重的胆汁酸吸收不良,摄入后7天只有1.6%的放射性标记胆汁酸保留。曾尝试使用胆汁酸隔离剂治疗,但由于副作用和对肠道维生素摄取无显著影响而停止。本病例强调了对不明原因脂溶性维生素缺乏症患者进行早期基因检测的重要性。这也强调了SLC10A2缺乏引起的原发性胆汁酸吸收不良的临床谱和处理需要进一步的研究。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Homozygosity for a variant in SLC10A2 and infancy onset severe fat-soluble vitamin deficiency due to bile acid malabsorption.

We present a case of a young female patient with persistent and severe fat-soluble vitamin deficiency since infancy. Despite extensive investigations during childhood, the underlying cause remained elusive. The patient was generally asymptomatic while receiving continuous vitamin subsidy. Exome sequencing performed at age 18 revealed a homozygous missense variant (Pro65Leu) in the gene SLC10A2, which encodes a bile acid transporter in the ileum. A bile retention scan showed severe bile acid malabsorption, with only 1.6% of radioactively labeled bile acids retained 7 days after intake. Treatment with a bile acid sequestrant was attempted but discontinued due to side effects and no significant effect on intestinal vitamin uptake. This case highlights the importance of early genetic testing in patients with unexplained fat-soluble vitamin deficiency. It also emphasizes the need for further research to elucidate the clinical spectrum and management of primary bile acid malabsorption due to SLC10A2 deficiency.

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