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杜兴氏和贝克氏肌营养不良症携带者风险女性对基因信息的掌控

Empowerment of genetic information by women at-risk of being carriers of Duchenne and Becker muscular dystrophies.

作者信息

Hoefel Alice Maria Luderitz, Weschenfelder Cesar Augusto, Rosa Bruna Faria, Donis Karina Carvalho, Saute Jonas Alex Morales

机构信息

Graduate Program in Medicine: Medical Sciences, Universidade Federal Do Rio Grande Do Sul, Porto Alegre, Brazil.

Medical Genetics Service, Hospital de Clínicas de Porto Alegre, Ramiro Barcelos, 2350, Porto Alegre, 90035-903, Brazil.

出版信息

J Community Genet. 2024 Apr;15(2):163-175. doi: 10.1007/s12687-023-00695-3. Epub 2024 Jan 2.

Abstract

The emergence of therapies acting on specific molecular targets for Duchenne and Becker muscular dystrophies (DBMD) led to expanded access of diagnostic DMD analysis. However, it is unclear how much of these advances have also improved healthcare and access to genetic testing for women at-risk of being carriers. This study evaluates the process of genetic counseling and empowerment of genetic information by women from DBMD families. We carried out a cross-sectional study between February and June 2022 in Brazil. The online survey with items regarding sociodemographic data; family history; access to health services; reproductive decisions; and the Genomic Outcome Scale was answered by 123 women recruited from a rare diseases reference service and a nationwide patient advocacy group. Genetic counseling was reported by 77/123 (62.6%) of women and 53.7% reported having performed genetic analysis of DMD. Although the majority knew about the risks for carriers of developing heart disease and muscle weakness, only 35% of potential carriers have had cardiac studies performed at least once in their lives. Country region, type of kinship, number of affected males in the family, age, notion of genetic risk, education level, and participation in advocacy groups were the main factors associated with adequate healthcare access to women and empowerment of genetic information. Education to health professionals and policies to expand access to carrier genetic testing, whether public policies or regulation of pharmaceutical companies' diagnostic programs, is paramount to improve the care of families with DBMD in Brazil.

摘要

针对杜兴氏和贝克氏肌肉营养不良症(DBMD)的特定分子靶点疗法的出现,使得杜兴氏肌营养不良症(DMD)诊断分析的可及性得到了扩展。然而,尚不清楚这些进展在多大程度上也改善了医疗保健状况以及为有携带者风险的女性提供基因检测的可及性。本研究评估了来自DBMD家庭的女性接受遗传咨询以及获取遗传信息的过程。2022年2月至6月期间,我们在巴西开展了一项横断面研究。123名从罕见病参考服务机构和全国性患者倡导组织招募的女性回答了关于社会人口统计学数据、家族病史、获得医疗服务的情况、生殖决策以及基因组结果量表的在线调查问卷。77/123(62.6%)的女性报告接受过遗传咨询,53.7%的女性报告进行过DMD基因分析。尽管大多数人知道携带者患心脏病和肌肉无力的风险,但只有35%的潜在携带者一生中至少进行过一次心脏检查。国家地区、亲属关系类型、家庭中受影响男性的数量、年龄、遗传风险观念、教育水平以及参与倡导组织是与女性获得充分医疗保健和获取遗传信息相关的主要因素。对卫生专业人员进行教育,并制定扩大携带者基因检测可及性的政策,无论是公共政策还是对制药公司诊断项目的监管,对于改善巴西DBMD家庭的护理至关重要。

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