Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report

Alexander disease is a form of leukoencephalopathy caused by mutations in the GFAP gene. There are three forms of the disease: infant, juvenile and adult. We present the clinical case of a patient born in 2004 (16 years old) with a debut of the disease at the age of 4 years with complex ticks. furth...

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Main Authors: A. I. Pavljuchkova, A. S. Kotov
Format: Article
Language:Russian
Published: ABV-press 2021-07-01
Series:Русский журнал детской неврологии
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Online Access:https://rjdn.abvpress.ru/jour/article/view/362
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author A. I. Pavljuchkova
A. S. Kotov
author_facet A. I. Pavljuchkova
A. S. Kotov
author_sort A. I. Pavljuchkova
collection DOAJ
description Alexander disease is a form of leukoencephalopathy caused by mutations in the GFAP gene. There are three forms of the disease: infant, juvenile and adult. We present the clinical case of a patient born in 2004 (16 years old) with a debut of the disease at the age of 4 years with complex ticks. further neurological symptoms progressed and appeared atactic gait, intention tremor by performing coordination tests, muscle hypotension, decreased tendon reflexes, nasal voices, and behavior changes.Magnetic resonance imaging revealed changes in the white matter of both frontal lobes. An analysis was made of 59 genes of the panel “Leukodystrophy/leukoencephalopathy” by the method of mass parallel sequencing on the Ion S5. A mutation of the GFAP gene (Nm_002055), 4 exon c.758C>A, p.ALA253Asp in a heterozygous state, not described in Human Gene mutation Database, was detected. The patient was confirmed to have a diagnosis of Alexander disease. According to tractography, a decrease in the number of fibers in the frontal lobes was found.The patient is currently receiving symptomatic treatment.
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spelling doaj-art-dae71c2e2820408a82f7cc49dee0eeb22025-08-20T03:44:13ZrusABV-pressРусский журнал детской неврологии2073-88032412-91782021-07-01161-2586210.17650/2073-8803-2021-16-1-2-58-62242Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case reportA. I. Pavljuchkova0A. S. Kotov1M.F. Vladimirsky Moscow Regional Research Clinical InstituteM.F. Vladimirsky Moscow Regional Research Clinical InstituteAlexander disease is a form of leukoencephalopathy caused by mutations in the GFAP gene. There are three forms of the disease: infant, juvenile and adult. We present the clinical case of a patient born in 2004 (16 years old) with a debut of the disease at the age of 4 years with complex ticks. further neurological symptoms progressed and appeared atactic gait, intention tremor by performing coordination tests, muscle hypotension, decreased tendon reflexes, nasal voices, and behavior changes.Magnetic resonance imaging revealed changes in the white matter of both frontal lobes. An analysis was made of 59 genes of the panel “Leukodystrophy/leukoencephalopathy” by the method of mass parallel sequencing on the Ion S5. A mutation of the GFAP gene (Nm_002055), 4 exon c.758C>A, p.ALA253Asp in a heterozygous state, not described in Human Gene mutation Database, was detected. The patient was confirmed to have a diagnosis of Alexander disease. According to tractography, a decrease in the number of fibers in the frontal lobes was found.The patient is currently receiving symptomatic treatment.https://rjdn.abvpress.ru/jour/article/view/362alexander diseasepreviously not described mutationmagnetic resonance imagingtractographygfap
spellingShingle A. I. Pavljuchkova
A. S. Kotov
Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
Русский журнал детской неврологии
alexander disease
previously not described mutation
magnetic resonance imaging
tractography
gfap
title Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
title_full Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
title_fullStr Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
title_full_unstemmed Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
title_short Juvenile form of alexander disease caused by a previously undescribed mutation in the GFAP gene. a case report
title_sort juvenile form of alexander disease caused by a previously undescribed mutation in the gfap gene a case report
topic alexander disease
previously not described mutation
magnetic resonance imaging
tractography
gfap
url https://rjdn.abvpress.ru/jour/article/view/362
work_keys_str_mv AT aipavljuchkova juvenileformofalexanderdiseasecausedbyapreviouslyundescribedmutationinthegfapgeneacasereport
AT askotov juvenileformofalexanderdiseasecausedbyapreviouslyundescribedmutationinthegfapgeneacasereport