Novel COL4A1 mutations identified in infants with congenital hemolytic anemia in association with brain malformations

Hum Genome Var. 2020 Nov 27;7(1):42. doi: 10.1038/s41439-020-00130-w.

Abstract

Genetic causes of undiagnosed hemolytic anemia in nineteen patients were analyzed by whole-exome sequencing, and novel COL4A1 variants were identified in four patients (21%). All patients were complicated with congenital malformations of the brain, such as porencephaly or schizencephaly. In these patients, hemolysis became less severe within 2 months after birth, and red cell transfusion was no longer required after 50 days, whereas chronic hemolysis continued.