Suppr超能文献

脊髓性肌萎缩症(SMA)结果不确定时的产前遗传咨询挑战

Prenatal genetic counseling challenges with indeterminate SMA results.

作者信息

Spangenberg Molly, Hendon Laura Godfrey, Goodloe Dana H, Brewer Fallon, Zhai Guihua, Gomes Alicia

机构信息

Department of Genetics, The University of Alabama at Birmingham, Birmingham, Alabama, USA.

Department of Pediatrics and Obstetrics and Gynecology, The University of Mississippi Medical Center, Jackson, Mississippi, USA.

出版信息

J Genet Couns. 2025 Apr;34(2):e2017. doi: 10.1002/jgc4.2017.

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular condition with complex genetic etiology. About 95% of individuals affected with this condition have a homozygous deletion of the SMN1 gene. One scenario that complicates risk is when a parent is identified as a possible silent carrier, meaning they have a [2 + 0] chromosome configuration. This configuration occurs when an individual has two copies of the SMN1 gene on one chromosome and no copies on the other chromosome. It is thought that 3.8-4.0% of the general population is a [2 + 0] carrier with a higher prevalence in African American and Hispanic populations. The [2 + 0] configuration makes it more difficult to calculate residual risk because testing cannot determine the difference between [2 + 0] carriers and [1 + 1] non-carriers, leading to indeterminate SMA carrier screening results. SMA was added to general population carrier screening in 2017, leading to an increase in the number of patients identified to have indeterminate results. Previous research has not examined how this addition has affected counseling practices involving indeterminate results. The purpose of this research was to gain a better understanding of the practices and challenges in this area, specifically within non-Ashkenazi Jewish (AJ) populations. This study utilized a quantitative survey with open-response questions. Responses from 49 prenatal genetic counselors from the United States and Canada were analyzed and it was found that genetic counselors face similar challenges when counseling indeterminate SMA results across all regions. These include negative patient emotions and both patient and referring provider misunderstanding, as highlighted in the qualitative data. Three major categories emerged including (1) challenges with patients, (2) challenges with referring providers, and (3) the effects of the 2017 addition to general population carrier screening. This study highlights the need for provider education surrounding indeterminate SMA results, the development of a visual aid, and future research from the patient and referring provider perspective.

摘要

脊髓性肌萎缩症(SMA)是一种常染色体隐性神经肌肉疾病,其遗传病因复杂。约95%受此病影响的个体存在SMN1基因的纯合缺失。一种使风险复杂化的情况是,当父母一方被确定为可能的沉默携带者时,即他们具有[2 + 0]染色体构型。当个体在一条染色体上有两份SMN1基因拷贝而在另一条染色体上没有拷贝时,就会出现这种构型。据认为,普通人群中有3.8 - 4.0%是[2 + 0]携带者,在非裔美国人和西班牙裔人群中的患病率更高。[2 + 0]构型使得计算残余风险更加困难,因为检测无法确定[2 + 0]携带者与[1 + 1]非携带者之间的差异,导致SMA携带者筛查结果不确定。SMA于2017年被纳入普通人群携带者筛查,导致被确定为结果不确定的患者数量增加。此前的研究尚未考察这一纳入对涉及不确定结果的咨询实践产生了怎样的影响。本研究的目的是更好地了解该领域的实践和挑战,特别是在非阿什肯纳兹犹太(AJ)人群中。本研究采用了带有开放式问题的定量调查。对来自美国和加拿大的49名产前遗传咨询师的回复进行了分析,发现遗传咨询师在为所有地区不确定的SMA结果提供咨询时面临类似的挑战。这些挑战包括患者的负面情绪以及患者和转诊医生的误解,定性数据中对此有突出体现。出现了三大类问题,包括(1)与患者相关的挑战,(2)与转诊医生相关的挑战,以及(3)2017年纳入普通人群携带者筛查的影响。本研究强调了围绕不确定的SMA结果对医疗服务提供者进行教育的必要性、开发一种视觉辅助工具以及从患者和转诊医生角度进行未来研究的必要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7ea5/11965976/322e9583a5f0/JGC4-34-0-g001.jpg

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验