Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report

Background: Metachromatic leukodystrophy (MLD) is an autosomal recessive hereditary neurodegenerative disease caused by a deficiency in arylsulfatase A (ARSA) activity and belongs to the group of lysosomal storage diseases. A biochemical diagnosis of MLD is based on determining th...

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Main Authors: Ekram Fateen, Zeinab Y. Abdallah
Format: Article
Language:English
Published: IMR Press 2024-12-01
Series:Frontiers in Bioscience-Scholar
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Online Access:https://www.imrpress.com/journal/FBS/16/4/10.31083/j.fbs1604020
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author Ekram Fateen
Zeinab Y. Abdallah
author_facet Ekram Fateen
Zeinab Y. Abdallah
author_sort Ekram Fateen
collection DOAJ
description Background: Metachromatic leukodystrophy (MLD) is an autosomal recessive hereditary neurodegenerative disease caused by a deficiency in arylsulfatase A (ARSA) activity and belongs to the group of lysosomal storage diseases. A biochemical diagnosis of MLD is based on determining the residual ARSA activity in leukocytes, skin fibroblasts, and urine. This study documents our biochemical experience and estimates the relative frequency of MLD over 21 years (2001–2022). Methods: This study analyzed 4357 suspected cases of MLD in Egypt. The ARSA activity was spectrophotometrically determined in leukocytes in all the referred cases. Results: Of these 4357 referred cases, 577 (13%) possessed decreased ARSA activity, less than 10% of the low normal range (50–200 micromole/gram protein/hour (μmol/g protein/h), and 104 cases were diagnosed as having a pseudodeficiency in enzyme activity (<20–50% of low–normal ARSA activity). The prevalence of MLD was 1.6/100,000. Conclusions: A diagnosis of MLD in Egypt is based on enzyme activity levels and clinical suspicion; molecular analysis was performed in a few cases.
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spelling doaj-art-825e8bd1692840828e5c250154622f6c2024-12-30T12:15:49ZengIMR PressFrontiers in Bioscience-Scholar1945-05162024-12-011642010.31083/j.fbs1604020S1945-0516(24)00117-5Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory ReportEkram Fateen0Zeinab Y. Abdallah1Biochemical Genetics Department, Human Genetics and Genome Research Institute, National Research Centre, 12622 Cairo, EgyptBiochemical Genetics Department, Human Genetics and Genome Research Institute, National Research Centre, 12622 Cairo, EgyptBackground: Metachromatic leukodystrophy (MLD) is an autosomal recessive hereditary neurodegenerative disease caused by a deficiency in arylsulfatase A (ARSA) activity and belongs to the group of lysosomal storage diseases. A biochemical diagnosis of MLD is based on determining the residual ARSA activity in leukocytes, skin fibroblasts, and urine. This study documents our biochemical experience and estimates the relative frequency of MLD over 21 years (2001–2022). Methods: This study analyzed 4357 suspected cases of MLD in Egypt. The ARSA activity was spectrophotometrically determined in leukocytes in all the referred cases. Results: Of these 4357 referred cases, 577 (13%) possessed decreased ARSA activity, less than 10% of the low normal range (50–200 micromole/gram protein/hour (μmol/g protein/h), and 104 cases were diagnosed as having a pseudodeficiency in enzyme activity (<20–50% of low–normal ARSA activity). The prevalence of MLD was 1.6/100,000. Conclusions: A diagnosis of MLD in Egypt is based on enzyme activity levels and clinical suspicion; molecular analysis was performed in a few cases.https://www.imrpress.com/journal/FBS/16/4/10.31083/j.fbs1604020metachromatic leukodystrophyarylsulfatase aautosomal recessiveenzyme activity
spellingShingle Ekram Fateen
Zeinab Y. Abdallah
Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
Frontiers in Bioscience-Scholar
metachromatic leukodystrophy
arylsulfatase a
autosomal recessive
enzyme activity
title Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
title_full Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
title_fullStr Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
title_full_unstemmed Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
title_short Relative Frequency of Metachromatic Leukodystrophy in Egypt: A Reference Laboratory Report
title_sort relative frequency of metachromatic leukodystrophy in egypt a reference laboratory report
topic metachromatic leukodystrophy
arylsulfatase a
autosomal recessive
enzyme activity
url https://www.imrpress.com/journal/FBS/16/4/10.31083/j.fbs1604020
work_keys_str_mv AT ekramfateen relativefrequencyofmetachromaticleukodystrophyinegyptareferencelaboratoryreport
AT zeinabyabdallah relativefrequencyofmetachromaticleukodystrophyinegyptareferencelaboratoryreport