Prenatal Clinical Findings in RASA1-Related Capillary Malformation-Arteriovenous Malformation Syndrome

Genes (Basel). 2023 Feb 22;14(3):549. doi: 10.3390/genes14030549.

Abstract

Pathogenic variants in RASA1 are typically associated with a clinical condition called "capillary malformation-arteriovenous malformation" (CM-AVM) syndrome, an autosomal dominant genetic disease characterized by a broad phenotypic variability, even within families. In CM-AVM syndrome, multifocal capillary and arteriovenous malformations are mainly localized in the central nervous system, spine and skin. Although CM-AVM syndrome has been widely described in the literature, only 21 cases with prenatal onset of clinical features have been reported thus far. Here, we report four pediatric cases of molecularly confirmed CM-AVM syndrome which manifested during the prenatal period. Polyhydramnios, non-immune hydrops fetalis and chylothorax are only a few possible aspects of this condition, but a correct interpretation of these prenatal signs is essential due to the possible fatal consequences of unrecognized encephalic and thoracoabdominal deep vascular malformations in newborns and in family members carrying the same RASA1 variant.

Keywords: RASA1; capillary malformation-arteriovenous malformation (CM-AVM); chylothorax; non-immune fetal hydrops; polyhydramnios; prenatal findings.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Arteriovenous Malformations* / diagnostic imaging
  • Arteriovenous Malformations* / genetics
  • Capillaries / abnormalities
  • Child
  • Female
  • GTPase-Activating Proteins / genetics
  • Humans
  • Infant, Newborn
  • Mutation
  • Port-Wine Stain* / diagnosis
  • Port-Wine Stain* / genetics
  • Port-Wine Stain* / pathology
  • Pregnancy
  • p120 GTPase Activating Protein / genetics

Substances

  • p120 GTPase Activating Protein
  • GTPase-Activating Proteins
  • RASA1 protein, human

Supplementary concepts

  • Capillary Malformation-Arteriovenous Malformation

Grants and funding

This work was supported, in part, by funding from Fondazione Bambino Gesù (Vite Coraggiose) and the Italian Ministry of Health (CCR-2017-23669081 to M.T., RF-2018-12366931 and GR-2019-12371203 to F.C.R.).