Neonatal Intensive Care Unit, Department of Pediatric Medicine, University Hospital of Saint-Étienne, 42055 Saint-Étienne, France.
Department of Clinical Genetics, Erasmus University Medical Center, PB 2040, 3000 CA Rotterdam, The Netherlands.
Clin Res Hepatol Gastroenterol. 2016 Dec;40(6):e65-e67. doi: 10.1016/j.clinre.2015.12.018. Epub 2016 Oct 4.
Congenital short-bowel syndrome (CSBS) is a rare neonatal pathology associated with poor prognosis and high mortality rate. We describe a newborn presenting CSBS intestinal malrotation and chronic intestinal pseudo-obstruction syndrome (CIPS), compound heterozygous for two previously unreported heterozygous mutations in Coxsackie and adenovirus receptor-like membrane protein (CLMP) gene, one in intron 1 (c.28+1G>C), the other on exon 4 (c502C>T, p.R168X). Both mutations are predicted to be pathogenic, leading to impaired splicing and the appearance of a premature stop codon, respectively. Our case is remarkable in that it concerns two heterozygous truncating mutations associated with a good clinical prognosis with a favorable cerebral and gastrointestinal outcome and a substantial enteral input at 8 months of age, despite a small intestine measuring only 35cm.
先天性短肠综合征(CSBS)是一种罕见的新生儿病理,预后不良,死亡率高。我们描述了一名患有 CSBS 肠旋转不良和慢性假性肠梗阻综合征(CIPS)的新生儿,其 Coxsackie 和腺病毒受体样膜蛋白(CLMP)基因存在两个先前未报道的杂合突变,一个位于内含子 1(c.28+1G>C),另一个位于外显子 4(c502C>T,p.R168X)。这两种突变均被预测为致病性的,分别导致剪接受损和出现提前终止密码子。我们的病例值得注意的是,它涉及两个杂合性截断突变,尽管小肠仅长 35cm,但在 8 个月大时,患儿具有良好的临床预后、良好的脑和胃肠道结局以及大量肠内输入。