Rehm Heidi L, Berg Jonathan S, Brooks Lisa D, Bustamante Carlos D, Evans James P, Landrum Melissa J, Ledbetter David H, Maglott Donna R, Martin Christa Lese, Nussbaum Robert L, Plon Sharon E, Ramos Erin M, Sherry Stephen T, Watson Michael S
From Harvard Medical School and Brigham and Women's Hospital and Partners HealthCare - all in Boston (H.L.R.); University of North Carolina, Chapel Hill (J.S.B., J.P.E.); National Human Genome Research Institute, National Institutes of Health (NIH) (L.D.B., E.M.R.), National Center for Biotechnology Information, National Library of Medicine, NIH (M.J.L., D.R.M., S.T.S.), and American College of Medical Genetics and Genomics (M.S.W.) - all in Bethesda, MD; Stanford University School of Medicine, Stanford (C.D.B.), and University of California, San Francisco, San Francisco (R.L.N.) - both in California; Geisinger Health System, Danville, PA (D.H.L., C.L.M.); and Baylor College of Medicine, Houston (S.E.P.).
N Engl J Med. 2015 Jun 4;372(23):2235-42. doi: 10.1056/NEJMsr1406261. Epub 2015 May 27.
On autopsy, a patient is found to have hypertrophic cardiomyopathy. The patient’s family pursues genetic testing that shows a “likely pathogenic” variant for the condition on the basis of a study in an original research publication. Given the dominant inheritance of the condition and the risk of sudden cardiac death, other family members are tested for the genetic variant to determine their risk. Several family members test negative and are told that they are not at risk for hypertrophic cardiomyopathy and sudden cardiac death, and those who test positive are told that they need to be regularly monitored for cardiomyopathy on echocardiography. Five years later, during a routine clinic visit of one of the genotype-positive family members, the cardiologist queries a database for current knowledge on the genetic variant and discovers that the variant is now interpreted as “likely benign” by another laboratory that uses more recently derived population-frequency data. A newly available testing panel for additional genes that are implicated in hypertrophic cardiomyopathy is initiated on an affected family member, and a different variant is found that is determined to be pathogenic. Family members are retested, and one member who previously tested negative is now found to be positive for this new variant. An immediate clinical workup detects evidence of cardiomyopathy, and an intracardiac defibrillator is implanted to reduce the risk of sudden cardiac death.
尸检时发现一名患者患有肥厚型心肌病。患者家属进行了基因检测,根据一项原始研究出版物中的研究,检测结果显示该病症存在“可能致病”的变异。鉴于该病症的显性遗传以及心源性猝死的风险,对其他家庭成员进行了该基因变异检测以确定他们的风险。几名家庭成员检测结果为阴性,并被告知他们没有患肥厚型心肌病和心源性猝死的风险,而检测结果为阳性的家庭成员则被告知需要定期通过超声心动图监测心肌病情况。五年后,在一名基因型阳性家庭成员的例行门诊就诊期间,心脏病专家查询数据库以获取有关该基因变异的最新知识,发现另一家使用更新得出的人群频率数据的实验室现在将该变异解释为“可能良性”。对一名受影响的家庭成员启动了一项新的针对与肥厚型心肌病相关的其他基因的检测面板,发现了另一种被确定为致病的变异。对家庭成员重新进行检测,一名之前检测为阴性的成员现在被发现对这种新变异呈阳性。立即进行的临床检查发现了心肌病的证据,并植入了心脏内除颤器以降低心源性猝死的风险。