TY - JOUR
T1 - SLC2A1 mutations are a rare cause of pediatric-onset hereditary spastic paraplegia
AU - Nicita, Francesco
AU - Schirinzi, Tommaso
AU - Stregapede, Fabrizia
AU - Vasco, Gessica
AU - Bertini, Enrico
AU - Travaglini, Lorena
N1 - Copyright © 2018 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.
PY - 2018/12/18
Y1 - 2018/12/18
N2 - SLC2A1 mutations cause glucose transporter type 1 deficiency syndrome, whose phenotypic spectrum is a continuum, ranging from classic to variant phenotypes, the latter accounting for about 10% of cases. Very few SLC2A1-mutated patients with a spastic paraplegia phenotype have been reported so far, and they are associated with paroxysmal choreo-athetosis (i.e., DYT9). The authors describe two sporadic children with pure and complex hereditary spastic paraplegia (HSP) without paroxysmal non-epileptic movement disorders harboring heterozygous de novo SLC2A1 pathogenic variants. These patients have been identified by a targeted panel for HSP among 140 pediatric- and adult-onset unrelated cases with pure and complex HSP, thus indicating an overall prevalence of 1.4% of SLC2A1 mutations, which increases to 3% if only pediatric-onset patients are considered. The implications of these findings in the diagnostic work-up of HSP patients are discussed.
AB - SLC2A1 mutations cause glucose transporter type 1 deficiency syndrome, whose phenotypic spectrum is a continuum, ranging from classic to variant phenotypes, the latter accounting for about 10% of cases. Very few SLC2A1-mutated patients with a spastic paraplegia phenotype have been reported so far, and they are associated with paroxysmal choreo-athetosis (i.e., DYT9). The authors describe two sporadic children with pure and complex hereditary spastic paraplegia (HSP) without paroxysmal non-epileptic movement disorders harboring heterozygous de novo SLC2A1 pathogenic variants. These patients have been identified by a targeted panel for HSP among 140 pediatric- and adult-onset unrelated cases with pure and complex HSP, thus indicating an overall prevalence of 1.4% of SLC2A1 mutations, which increases to 3% if only pediatric-onset patients are considered. The implications of these findings in the diagnostic work-up of HSP patients are discussed.
U2 - 10.1016/j.ejpn.2018.12.004
DO - 10.1016/j.ejpn.2018.12.004
M3 - Article
C2 - 30616884
JO - European Journal of Paediatric Neurology
JF - European Journal of Paediatric Neurology
SN - 1090-3798
ER -