Suppr超能文献

迟发性法布里病:心脏后遗症。

Late-onset Fabry disease: the cardiac sequela.

机构信息

Internal Medicine, Albany Medical Center, Albany, New York, USA

Department of Cardiology, Albany Medical Center, Albany, New York, USA.

出版信息

BMJ Case Rep. 2022 Jun 9;15(6):e247917. doi: 10.1136/bcr-2021-247917.

Abstract

We describe a patient with Fabry disease (FD) who initially presented with atrial fibrillation without left ventricular hypertrophy (LVH) 14 years before being correctly diagnosed with FD. In the interim, he survived a myocardial infarction complicated by ventricular fibrillation, and his severe LVH was misdiagnosed as sarcomeric hypertrophic cardiomyopathy. In the following 4 years, he developed proteinuric kidney disease, neuropathy, sensorineural hearing loss and gastrointestinal symptoms. The patient was eventually readmitted for an overt heart failure (HF) exacerbation and was seen by an HF cardiologist. The constellation of systemic findings led to further diagnostic testing, including an endomyocardial biopsy, tests to determine alpha-galactosidase A enzyme activity and α-galactosidase A gene (GLA) analysis. The results of the patient's tests were consistent with FD and he was started on enzyme replacement therapy. To our knowledge, this is the first detailed description of a late-onset phenotype of FD with c.146 G>C GLA variant. In addition, this case serves as a potent reminder to pay meticulous attention to 'red flags' accompanying LVH.

摘要

我们描述了一位 Fabry 病(FD)患者,他在被正确诊断为 FD 之前 14 年前首次出现心房颤动而无左心室肥厚(LVH)。在此期间,他曾经历过一次心肌梗死并发心室颤动,他的严重 LVH 被误诊为肌节性肥厚型心肌病。在接下来的 4 年中,他出现了蛋白尿性肾病、神经病、感觉神经性听力损失和胃肠道症状。最终,他因明显的心衰(HF)加重而再次入院,并由 HF 心脏病专家就诊。全身症状的综合表现促使进行了进一步的诊断性检查,包括心内膜心肌活检、α-半乳糖苷酶 A 酶活性和α-半乳糖苷酶 A 基因(GLA)分析测试。患者的检查结果与 FD 一致,随后开始接受酶替代治疗。据我们所知,这是首例伴有 c.146 G>C GLA 变异的 FD 迟发性表型的详细描述。此外,该病例有力地提醒我们要细致关注伴有 LVH 的“危险信号”。

相似文献

1
Late-onset Fabry disease: the cardiac sequela.迟发性法布里病:心脏后遗症。
BMJ Case Rep. 2022 Jun 9;15(6):e247917. doi: 10.1136/bcr-2021-247917.

本文引用的文献

2
Global research on Fabry's disease: Demands for a rare disease.法布里病的全球研究:对罕见病的需求。
Mol Genet Genomic Med. 2020 Sep;8(9):e1163. doi: 10.1002/mgg3.1163. Epub 2020 Feb 7.

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