Epidemiology, Diagnostics, and Management of Vein of Galen Malformation

Background: Vein of Galen malformation (VGM) is a rare congenital intracerebral vascular malformation. The aim of this study was to gain reliable data about epidemiology; clinical manifestations; and pre-, peri- and postinterventional treatment strategies in Germany. Methods: This prospective epidemiologic study was conducted by Erhebungseinheit fur Seltene Padiatrische Erkrankungen in Deutschland, a hospital-based German nationwide surveillance unit for rare pediatric diseases. Patients aged upto 18 years with a new diagnosis of VGM were prospectively included between January 1, 2014 and December 31, 2015. Results: In the two-year study period, 30 individuals with newly diagnosed VGM were identified, resulting in an estimated annual incidence rate of VGM in Germany of at least 1: 58,100 live births. The main symptoms were age dependent. Neonates presented with mainly cardiac manifestations of high output failure; infants and older children mainly suffered from neurological complications. Of the patients with endovascular treatment 95% survived, but only 41% of these patients were discharged home without any complications. The Bic<^>etre Neonatal Evaluation Score was a good predictor for poor outcome but could have led to palliative care in some patients with good outcome. Conclusions: The incidence of VGM in Germany is estimated to be 1:58,100 live births. The majority of patients were eligible for endovascular treatment. Nevertheless, due to side effects and long-term sequelae in the majority of patients, further improvement in care of children with VGM is needed. (C) 2021 Elsevier Inc. All rights reserved.

Automated summary

Vein of Galen malformation (VGM) is a rare congenital intracerebral vascular malformation. This study in Germany estimated an annual incidence rate of VGM to be 1:58,100 live births, with patients presenting different clinical manifestations based on their age. Endovascular treatment showed high survival rates, but some patients still experienced complications after discharge. Further improvement in care for children with VGM is necessary due to side effects and long-term sequelae.