高级检索
当前位置: 首页 > 详情页

Clinical characterization and diagnosis of cystic fibrosis through exome sequencing in Chinese infants with Bartter-syndrome-like hypokalemia alkalosis

文献详情

资源类型:
WOS体系:
Pubmed体系:

收录情况: ◇ SCIE ◇ 统计源期刊 ◇ CSCD-C

单位: [1]Huazhong Univ Sci & Technol, Tongji Hosp, Dept Pediat, Tongji Med Coll, Wuhan 430030, Hubei, Peoples R China
出处:
ISSN:

关键词: cystic fibrosis pseudo-Bartter syndrome hypokalemic alkalosis CFTR gene mutations infants diagnosis

摘要:
Cystic fibrosis (CF) is a fatal autosomal-recessive disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. CF is characterized by recurrent pulmonary infection with obstructive pulmonary disease. CF is common in the Caucasian population but is rare in the Chinese population. The symptoms of early-stage CF are often untypical and may sometimes manifest as Bartter syndrome (BS)-like hypokalemic alkalosis. Therefore, the ability of doctors to differentiate CF from BS-like hypokalemic alkalosis in Chinese infants is a great challenge in the timely and accurate diagnosis of CF. In China, sporadic CF has not been diagnosed in children younger than three years of age to date. Three infants, who were initially admitted to our hospital over the period of June 2013 to September 2014 with BS-like hypokalemic alkalosis, were diagnosed with CF through exome sequencing and sweat chloride measurement. The compound heterozygous mutations of the CFTR gene were detected in two infants, and a homozygous missense mutation was found in one infant. Among the six identified mutations, two are novel point mutations (c.1526G > C and c.3062C > T) that are possibly pathogenic. The three infants are the youngest Chinese patients to have been diagnosed with sporadic CF at a very early stage. Follow-up examination showed that all of the cases remained symptom-free after early intervention, indicating the potential benefit of very early diagnosis and timely intervention in children with CF. Our results demonstrate the necessity of distinguishing CF from BS in Chinese infants with hypokalemic alkalosis and the significant diagnostic value of powerful exome sequencing for rare genetic diseases. Furthermore, our findings expand the CFTR mutation spectrum associated with CF.

语种:
被引次数:
WOS:
PubmedID:
中科院(CAS)分区:
出版当年[2017]版:
大类 | 4 区 医学
小类 | 4 区 医学:研究与实验 4 区 肿瘤学
最新[2025]版:
大类 | 2 区 医学
小类 | 2 区 医学:内科 2 区 医学:研究与实验 2 区 肿瘤学
JCR分区:
出版当年[2016]版:
Q3 MEDICINE, RESEARCH & EXPERIMENTAL Q4 ONCOLOGY
最新[2023]版:
Q2 MEDICINE, RESEARCH & EXPERIMENTAL Q2 ONCOLOGY

影响因子: 最新[2023版] 最新五年平均 出版当年[2016版] 出版当年五年平均 出版前一年[2015版] 出版后一年[2017版]

第一作者:
第一作者单位: [1]Huazhong Univ Sci & Technol, Tongji Hosp, Dept Pediat, Tongji Med Coll, Wuhan 430030, Hubei, Peoples R China
通讯作者:
推荐引用方式(GB/T 7714):
APA:
MLA:

资源点击量:591 今日访问量:0 总访问量:443 更新日期:2025-06-01 建议使用谷歌、火狐浏览器 常见问题

版权所有:重庆聚合科技有限公司 渝ICP备12007440号-3 地址:重庆市两江新区泰山大道西段8号坤恩国际商务中心16层(401121)