Medical Genetics Branch, National Human Genome Research Institute, Bethesda, Maryland, USA.
Am J Med Genet A. 2012 Dec;158A(12):3087-100. doi: 10.1002/ajmg.a.35638. Epub 2012 Nov 19.
VACTERL association (sometimes termed "VATER association" depending on which component features are included) is typically defined by the presence of at least three of the following congenital malformations, which tend to statistically co-occur in affected individuals: Vertebral anomalies, Anal atresia, Cardiac malformations, Tracheo-Esophageal fistula, Renal anomalies, and Limb abnormalities. Although the clinical criteria for VACTERL association may appear to be straightforward, there is wide variability in the way clinical geneticists define the disorder and the genetic testing strategy they use when confronted with an affected patient. In order to describe this variability and determine the most commonly used definitions and testing modalities, we present the results of survey responses by 121 clinical geneticists. We discuss the results of the survey responses, provide a literature review and commentary from a group of physicians who are currently involved in clinical and laboratory-based research on VACTERL association, and offer an algorithm for genetic testing in patients with this association.
VACTERL 协会(有时根据包含的哪些特征成分而被称为“VATER 协会”)通常定义为存在以下至少三种先天性畸形,这些畸形在受影响的个体中往往会同时出现:脊柱异常、肛门闭锁、心脏畸形、气管食管瘘、肾脏异常和肢体异常。尽管 VACTERL 协会的临床标准似乎很直接,但临床遗传学家在定义该疾病以及在面对受影响的患者时使用的基因检测策略方面存在很大的差异。为了描述这种可变性并确定最常用的定义和检测方式,我们展示了 121 名临床遗传学家的调查回复结果。我们讨论了调查回复的结果,提供了目前参与 VACTERL 协会的临床和基于实验室的研究的一组医生的文献综述和评论,并为患有这种协会的患者提供了基因检测的算法。