Aetiology and management of hereditary aortopathy

Nat Rev Cardiol. 2017 Apr;14(4):197-208. doi: 10.1038/nrcardio.2016.211. Epub 2017 Jan 19.

Abstract

Aortic aneurysms are a major health problem because they account for 1-2% of all deaths in the Western population. Although abdominal aortic aneurysms (AAAs) are more prevalent than thoracic aortic aneurysms (TAAs), TAAs have been more exhaustively studied over the past 2 decades because they have a higher heritability and affect younger individuals. Gene identification in both syndromic and nonsyndromic TAA is proceeding at a rapid pace and has already pinpointed >20 genes associated with familial TAA risk. Whereas these genes explain <30% of all cases of familial TAA, their functional characterization has substantially improved our knowledge of the underlying pathological mechanisms. As such, perturbed extracellular matrix homeostasis, transforming growth factor-β signalling, and vascular smooth muscle cell contractility have been proposed as important processes in TAA pathogenesis. These new insights enable novel treatment options that are currently being investigated in large clinical trials. Moreover, together with the advent of next-generation sequencing approaches, these genetic findings are promoting a shift in the management of patients with TAA by enabling gene-tailored interventions. In this Review, we comprehensively describe the molecular landscape of familial TAA, and we discuss whether familial TAA, from a biological point of view, can serve as a paradigm for the genetically more complex forms of the condition, such as sporadic TAA or AAA.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aortic Aneurysm, Thoracic* / diagnosis
  • Aortic Aneurysm, Thoracic* / genetics
  • Aortic Aneurysm, Thoracic* / metabolism
  • Aortic Aneurysm, Thoracic* / therapy
  • Extracellular Matrix / metabolism*
  • Humans
  • Multifactorial Inheritance
  • Muscle, Smooth, Vascular / metabolism*
  • Patient Selection
  • Risk Assessment
  • Signal Transduction / physiology