Liu Yun-Feng, Han Tong-Yan, Tong Xiao-Mei, Wang Jing, Tang Ya-Nan, Cui Li-Gang, Zhu Xiao-Hui, Piao Mei-Hua, Wang Qing-Qing, Wu Hui
Department of Pediatrics, Peking University Third Hospital, Beijing 100191, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2018 Nov;20(11):939-943. doi: 10.7499/j.issn.1008-8830.2018.11.012.
A boy aged 2 months (born at 36 weeks of gestation) was admitted due to cough and dyspnea. After admission, he was found to have persistent hypertension, proteinuria, and persistent convulsion, and imaging examination showed extensive calcification of the aorta and major branches and stenosis of local lumens of the abdominal aorta and the right renal artery with increased blood flow velocity. The boy was admitted during the neonatal period due to wet lung and pulmonary arterial hypertension and was found to have hypertension and proteinuria. High-throughput whole-exome sequencing was performed and found two compound heterozygous mutations in the ENPP1 gene from his parents, c.130C>T (p.Q44X) and c.1112A>T (p.Y371F). c.130C>T was a nonsense mutation, which could cause partial deletion of protein from 44 amino acids, and was defined as a primary pathogenic mutation. c.1112A>T was a missense mutation which had been reported as a pathogenic mutation associated with idiopathic infantile arterial calcification (IIAC). Therefore, he was diagnosed with IIAC. He was given phosphonate drugs, antihypertensive drugs, anticonvulsion treatment, and respiratory support. Blood pressure was maintained at the upper limit of normal value. There was no deterioration of arterial calcification. It is concluded that IIAC should be considered for infants with persistent hypertension and extensive vascular calcification, and imaging and genetic examinations should be performed as early as possible to make a confirmed diagnosis.
一名2个月大的男孩(孕36周出生)因咳嗽和呼吸困难入院。入院后,发现他患有持续性高血压、蛋白尿和持续性惊厥,影像学检查显示主动脉及主要分支广泛钙化,腹主动脉和右肾动脉局部管腔狭窄,血流速度增加。该男孩在新生儿期因湿肺和肺动脉高压入院,当时就发现有高血压和蛋白尿。进行了高通量全外显子组测序,发现其父母的ENPP1基因存在两个复合杂合突变,即c.130C>T(p.Q44X)和c.1112A>T(p.Y371F)。c.130C>T是一个无义突变,可导致蛋白质从第44个氨基酸处部分缺失,被定义为主要致病突变。c.1112A>T是一个错义突变,已被报道为与特发性婴儿动脉钙化(IIAC)相关的致病突变。因此,他被诊断为IIAC。给予他膦酸盐药物、抗高血压药物、抗惊厥治疗及呼吸支持。血压维持在正常上限。动脉钙化没有恶化。结论是,对于患有持续性高血压和广泛血管钙化的婴儿应考虑IIAC,应尽早进行影像学和基因检查以确诊。