Original article
Cortical malformations and COL4A1 mutation:
Three new cases
G. Vitale
a
, A. Pichiecchio
a,b,*
, F. Ormitti
c
, D. Tonduti
d
, A. Asaro
e
,
L. Farina
b
, B. Piccolo
f
, A. Percesepe
g
, S. Bastianello
a,b
, S. Orcesi
a,h
,
COL4A1 International Study Group
a
Department of Brain and Behavioral Sciences, University of Pavia, Pavia, Italy
b
Department of Neuroradiology, IRCCS Mondino Foundation, Pavia, Italy
c
Neuroradiology Unit, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy
d
Child Neurology Unit, V. Buzzi Children's Hospital, Milan, Italy
e
Genomic and Post-Genomic Center, IRCCS Mondino Foundation, Pavia, Italy
f
Child Neuropsychiatry Unit, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy
g
Medical Genetics, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy
h
Child and Adolescence Neurology Unit, IRCCS Mondino Foundation, Pavia, Italy
article info
Article history:
Received 25 September 2018
Received in revised form
13 February 2019
Accepted 17 February 2019
abstract
Aim: The COL4A1 gene (13q34) encodes the a1 chain of type IV collagen, a crucial compo-
nent of the basal membrane. COL4A1 mutations have been identified as a cause of a
multisystem disease.
Brain MRI in COL4A1-mutated patients typically shows vascular abnormalities and
white matter lesions. Cortical malformations (specifically schizencephaly) have also
recently been described in these patients, suggesting that these, too, could be part of the
phenotypic spectrum of COL4A1 mutations.
The aim of our work was to retrospectively evaluate COL4A1-mutated subjects diag-
nosed at our centers in order to assess the frequency and define the type of cortical mal-
formations encountered in these individuals.
Method: We retrospectively reviewed MRI data of 18 carriers of COL4A1 mutations diag-
nosed in our centers between 2010 and 2016.
Results: We identified polymicrogyria in two patients, and schizencephaly in the mother of
a further patient.
Interpretation: Our findings confirm that cortical malformations should be considered to fall
within the phenotypic spectrum of COL4A1 mutations and show that not only schizence-
phaly but also polymicrogyria can also be found in mutated individuals. Although further
studies are needed to clarify the underlying pathogenetic mechanism, independently of this,
the timing of the brain damage could be the crucial factor determining the type of lesion.
© 2019 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights
reserved.
* Corresponding author. Via Mondino 2, 27100, Pavia, Italy.
E-mail address: anna.pichiecchio@mondino.it (A. Pichiecchio).
Official Journal of the European Paediatric Neurology Society
european journal of paediatric neurology xxx (xxxx) xxx
https://doi.org/10.1016/j.ejpn.2019.02.006
1090-3798/© 2019 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.
Please cite this article as: Vitale G et al., Cortical malformations and COL4A1 mutation: Three new cases, European Journal of Paediatric
Neurology, https://doi.org/10.1016/j.ejpn.2019.02.006