RECQL4 相关综合征的非典型表现。

IF 2.4 3区 医学 Q2 HEMATOLOGY
Liron D Grossman, Sarah Baldino, Kristin Zelley, Frank Balis, Rochelle Bagatell, Jennifer M Kalish, Suzanne P MacFarland
{"title":"RECQL4 相关综合征的非典型表现。","authors":"Liron D Grossman, Sarah Baldino, Kristin Zelley, Frank Balis, Rochelle Bagatell, Jennifer M Kalish, Suzanne P MacFarland","doi":"10.1002/pbc.31315","DOIUrl":null,"url":null,"abstract":"<p><p>RECQL4-related syndromes are a group of rare cancer-predisposition syndromes caused by biallelic pathogenic/likely pathogenic variants (PV/LPV) in the DNA helicase gene, RECQL4. Genetic testing is typically prompted by the presence of one or more hallmark clinical features, and in the absence of such manifestations, diagnosis may be delayed or even missed. We describe five patients with biallelic germline mutations in RECQL4 who presented atypically, without the hallmark clinical manifestations of this syndrome. Three of these patients developed osteosarcoma, underscoring the importance of recognizing atypical presentations of Rothmund-Thomson syndrome (RTS) to allow for early awareness and surveillance for cancer.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":null,"pages":null},"PeriodicalIF":2.4000,"publicationDate":"2024-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Atypical presentations of RECQL4-related syndromes.\",\"authors\":\"Liron D Grossman, Sarah Baldino, Kristin Zelley, Frank Balis, Rochelle Bagatell, Jennifer M Kalish, Suzanne P MacFarland\",\"doi\":\"10.1002/pbc.31315\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>RECQL4-related syndromes are a group of rare cancer-predisposition syndromes caused by biallelic pathogenic/likely pathogenic variants (PV/LPV) in the DNA helicase gene, RECQL4. Genetic testing is typically prompted by the presence of one or more hallmark clinical features, and in the absence of such manifestations, diagnosis may be delayed or even missed. We describe five patients with biallelic germline mutations in RECQL4 who presented atypically, without the hallmark clinical manifestations of this syndrome. Three of these patients developed osteosarcoma, underscoring the importance of recognizing atypical presentations of Rothmund-Thomson syndrome (RTS) to allow for early awareness and surveillance for cancer.</p>\",\"PeriodicalId\":19822,\"journal\":{\"name\":\"Pediatric Blood & Cancer\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.4000,\"publicationDate\":\"2024-09-24\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Blood & Cancer\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1002/pbc.31315\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Blood & Cancer","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/pbc.31315","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

RECQL4相关综合征是一组罕见的癌症易感综合征,由DNA螺旋酶基因RECQL4的双拷贝致病/可能致病变体(PV/LPV)引起。基因检测通常是在出现一个或多个标志性临床特征时进行的,如果没有这些表现,诊断可能会被延迟甚至漏诊。我们描述了五名 RECQL4 基因双倍性种系突变的患者,他们的症状不典型,没有该综合征的标志性临床表现。这些患者中有三人患上了骨肉瘤,这突出了识别罗斯蒙特-汤姆森综合征(RTS)非典型表现的重要性,以便及早发现和监测癌症。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Atypical presentations of RECQL4-related syndromes.

RECQL4-related syndromes are a group of rare cancer-predisposition syndromes caused by biallelic pathogenic/likely pathogenic variants (PV/LPV) in the DNA helicase gene, RECQL4. Genetic testing is typically prompted by the presence of one or more hallmark clinical features, and in the absence of such manifestations, diagnosis may be delayed or even missed. We describe five patients with biallelic germline mutations in RECQL4 who presented atypically, without the hallmark clinical manifestations of this syndrome. Three of these patients developed osteosarcoma, underscoring the importance of recognizing atypical presentations of Rothmund-Thomson syndrome (RTS) to allow for early awareness and surveillance for cancer.

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
Pediatric Blood & Cancer
Pediatric Blood & Cancer 医学-小儿科
CiteScore
4.90
自引率
9.40%
发文量
546
审稿时长
1.5 months
期刊介绍: Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.
×
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