The importance of geographic and sociodemographic aspects in the characterization of mucopolysaccharidoses: a case series from Ceará state (Northeast Brazil)

Author:

Santos Augusto1,Mariath Luiza Monteavaro1,Trapp Franciele2,Facchin Ana Carolina Brusius2,Leistner-Segal Sandra2,Kubaski Francyne2,Giugliani Roberto1,Schuler-Faccini Lavinia1,Ribeiro Erlane Marques3

Affiliation:

1. Instituto Nacional de Ciência e Tecnologia de Genética Médica Populacional (INaGeMP)

2. Hospital de Clínicas de Porto Alegre

3. Centro Universitário Christus (Unichristus)

Abstract

Abstract

Geographic and sociodemographic aspects may influence the natural history and epidemiology of mucopolysaccharidoses (MPS). In this case series, we have evaluated clinical and molecular data from 76 individuals with MPS from a region in Brazilian Northeast. MPS II was the most common type, with the most affected individuals presenting missense pathogenic variants. Patients with MPS I proved to be the most severe clinical phenotype, presenting the first symptoms (mean: 7.1 months; SD = 4.5) and being diagnosed earlier (2.2 years; SD = 2.1) in comparison with the other types. In addition, we have shown that 13 individuals with MPS VI were born of consanguineous marriages in small, nearby cities, in a place where geographical isolation, consanguinity, and clusters of genetic diseases were previously reported. Ten of these individuals (at least, seven different families) presented a rare pathogenic variant in the ARSB gene, c.1143-8T > G in homozygosity, previously reported only among Iberian and South American patients. We discuss the possible evolutionary processes and biosocial dynamics that can help to explain this finding in terms of population medical genetics and public health.

Publisher

Springer Science and Business Media LLC

Reference51 articles.

1. Molecular analysis of the CTSK gene in a cohort of 33 Brazilian families with pycnodysostosis from a cluster in a Brazilian Northeast region;Araujo TF;Eur J Med Res,2016

2. A large family with CYLD cutaneous syndrome: medical genetics at the community level;Arruda AP;J Community Genet,2020

3. The natural history of MPS I: global perspectives from the MPS I Registry;Beck M;Genet Sci,2014

4. Bonilla C, Sortica VA, Schuler-Faccini L, Matijasevich A, Scheffer MC (2021) Medical genetics workforce in Brazil: practitioners, services, and disease distribution. MedRxiv, 2021.10.14.21265027. https://doi.org/10.1101/2021.10.14.21265027

5. Phenotype-oriented NGS panels for mucopolysaccharidoses: Validation and potential use in the diagnostic flowchart;Brusius-Facchin AC;Genet Mol Biology,2019

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3