什么是囊性纤维化筛查阳性不确诊?什么不是?

IF 1.1 4区 医学 Q3 PEDIATRICS
Emily Devoy, Dominic Hughes, Asma Falah Alharbi, Jacqueline Francis, Jane C Davies
{"title":"什么是囊性纤维化筛查阳性不确诊?什么不是?","authors":"Emily Devoy, Dominic Hughes, Asma Falah Alharbi, Jacqueline Francis, Jane C Davies","doi":"10.1136/archdischild-2023-326767","DOIUrl":null,"url":null,"abstract":"<p><p>Since screening for cystic fibrosis (CF) was incorporated into the newborn screening program, the number of recognised variants in the <i>CF transmembrane conductance regulator (CFTR</i>) gene has significantly increased. This has led to the discovery of combinations of gene variants with an uncertain prognosis. One outcome is the designation of 'cystic fibrosis screen positive inconclusive diagnosis' (CFSPID). While the majority of these children are expected to be unaffected by their <i>CFTR</i> variants, a small proportion have been seen to develop symptoms or increasing sweat chloride levels over time, which may reflect dysfunction of the CFTR protein.As the number of children with CFSPID increases, paediatricians and those working in primary care are more likely to encounter them in their practice. It is important that professionals have an understanding of CFSPID: what it is and, importantly, what it is not (ie, they do not have CF). In this article, we hope to explore this using some example cases, illustrating the ways in which these children may present symptomatically and how to manage them.</p>","PeriodicalId":55471,"journal":{"name":"Archives of Disease in Childhood-Education and Practice Edition","volume":" ","pages":"237-241"},"PeriodicalIF":1.1000,"publicationDate":"2024-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"What is cystic fibrosis screen positive inconclusive diagnosis? And what is it not?\",\"authors\":\"Emily Devoy, Dominic Hughes, Asma Falah Alharbi, Jacqueline Francis, Jane C Davies\",\"doi\":\"10.1136/archdischild-2023-326767\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Since screening for cystic fibrosis (CF) was incorporated into the newborn screening program, the number of recognised variants in the <i>CF transmembrane conductance regulator (CFTR</i>) gene has significantly increased. This has led to the discovery of combinations of gene variants with an uncertain prognosis. One outcome is the designation of 'cystic fibrosis screen positive inconclusive diagnosis' (CFSPID). While the majority of these children are expected to be unaffected by their <i>CFTR</i> variants, a small proportion have been seen to develop symptoms or increasing sweat chloride levels over time, which may reflect dysfunction of the CFTR protein.As the number of children with CFSPID increases, paediatricians and those working in primary care are more likely to encounter them in their practice. It is important that professionals have an understanding of CFSPID: what it is and, importantly, what it is not (ie, they do not have CF). In this article, we hope to explore this using some example cases, illustrating the ways in which these children may present symptomatically and how to manage them.</p>\",\"PeriodicalId\":55471,\"journal\":{\"name\":\"Archives of Disease in Childhood-Education and Practice Edition\",\"volume\":\" \",\"pages\":\"237-241\"},\"PeriodicalIF\":1.1000,\"publicationDate\":\"2024-09-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Archives of Disease in Childhood-Education and Practice Edition\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1136/archdischild-2023-326767\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Archives of Disease in Childhood-Education and Practice Edition","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1136/archdischild-2023-326767","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0

摘要

自囊性纤维化(CF)筛查被纳入新生儿筛查计划以来,CF 跨膜传导调节器(CFTR)基因中可识别的变异体数量大幅增加。这导致了预后不确定的基因变异组合的发现。其中一个结果就是 "囊性纤维化筛查阳性诊断不确定"(CFSPID)。虽然这些儿童中的大多数预计不会受到 CFTR 变异的影响,但也有一小部分儿童随着时间的推移出现症状或汗液氯化物水平升高,这可能反映了 CFTR 蛋白的功能障碍。随着 CFSPID 患儿人数的增加,儿科医生和初级保健人员更有可能在实践中遇到他们。专业人员必须了解 CFSPID:它是什么,重要的是,它不是什么(即他们没有 CF)。在本文中,我们希望通过一些案例来探讨这一问题,说明这些儿童可能出现症状的方式以及如何处理这些症状。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
What is cystic fibrosis screen positive inconclusive diagnosis? And what is it not?

Since screening for cystic fibrosis (CF) was incorporated into the newborn screening program, the number of recognised variants in the CF transmembrane conductance regulator (CFTR) gene has significantly increased. This has led to the discovery of combinations of gene variants with an uncertain prognosis. One outcome is the designation of 'cystic fibrosis screen positive inconclusive diagnosis' (CFSPID). While the majority of these children are expected to be unaffected by their CFTR variants, a small proportion have been seen to develop symptoms or increasing sweat chloride levels over time, which may reflect dysfunction of the CFTR protein.As the number of children with CFSPID increases, paediatricians and those working in primary care are more likely to encounter them in their practice. It is important that professionals have an understanding of CFSPID: what it is and, importantly, what it is not (ie, they do not have CF). In this article, we hope to explore this using some example cases, illustrating the ways in which these children may present symptomatically and how to manage them.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
1.70
自引率
0.00%
发文量
68
审稿时长
>12 weeks
期刊介绍: Archives of Disease in Childhood is an international peer review journal that aims to keep paediatricians and others up to date with advances in the diagnosis and treatment of childhood diseases as well as advocacy issues such as child protection. It focuses on all aspects of child health and disease from the perinatal period (in the Fetal and Neonatal edition) through to adolescence. ADC includes original research reports, commentaries, reviews of clinical and policy issues, and evidence reports. Areas covered include: community child health, public health, epidemiology, acute paediatrics, advocacy, and ethics.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信