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IFITM5 丝氨酸 40 亮氨酸变异体可表现为产前卡斐骨病。

The IFITM5 Ser40Leu variant can manifest as prenatal Caffey disease.

机构信息

Nephrology Service, Department of Pediatrics, KK Women's and Children's Hospital, Singapore, Singapore.

Pediatric Academic Clinical Programme, Duke-NUS Medical School, Singapore, Singapore.

出版信息

Am J Med Genet A. 2024 Feb;194(2):358-362. doi: 10.1002/ajmg.a.63420. Epub 2023 Oct 6.

Abstract

We report on a female neonate with a clinico-radiological presentation in keeping with a lethal form of prenatal Caffey disease (PCH). She had antenatal and postnatal features of severely bowed long bones, small chest, diaphyseal hyperostosis and polyhydramnios and died shortly after birth. Initial testing excluded COL1A1-related PCH, as an OI gene panel, consisting of COL1A1, COL1A2, CRTAP, and P3H1 genes, was negative. Targeted sequencing using a gene panel was performed and a de novo heterozygous, likely pathogenic variant in IFITM5: c.119C > T(p.Ser40Leu) was identified, which was previously described to cause a severe form of progressively deforming osteogenesis imperfect (OI). To our knowledge, variants in IFITM5 have not been reported in infantile Caffey disease (ICH) or PCH. Given that the pathogenesis of PCH is largely unknown, we postulate that a subset of PCH may be associated with variants in IFITM5.

摘要

我们报告了一例女性新生儿,其临床表现和影像学检查符合产前 Caffey 病(PCH)的致死形式。她具有严重弯曲的长骨、小胸廓、骨干骨过度增生和羊水过多的产前和产后特征,并在出生后不久死亡。初步检测排除了 COL1A1 相关的 PCH,因为 OI 基因panel(包含 COL1A1、COL1A2、CRTAP 和 P3H1 基因)检测结果为阴性。随后进行了靶向测序基因panel 检测,发现 IFITM5 中存在一个新发杂合的、可能致病的变异:c.119C>T(p.Ser40Leu),该变异先前被描述可导致严重进行性骨发育不全(OI)。据我们所知,IFITM5 的变异尚未在婴儿 Caffey 病(ICH)或 PCH 中报道。鉴于 PCH 的发病机制尚不清楚,我们推测,PCH 的一部分可能与 IFITM5 的变异有关。

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