Franciele Barbosa Trapp, Mariluce Riegel, Kristiane Michelin-Tirelli, Layzon Antonio Lemos da Silva, Gabrielle Dineck Iop, Fernanda Bender-Pasetto, Fernanda Medeiros Sebastião, Inamara da Silva Moraes, Franciele Fátima Lopes, Osmar Rachor Toledo Dos Santos, Fernanda da Rosa Pereira, Laysla Pedelhes Silva, Andrius Anselmi Luiz, Cleiton Amaral da Silva, Karen Daniela Piccoli Lucas, Alice Brinckmann Oliveira Netto, Carolina Serpa Brasil, Sandra Leistner, Ana Carolina Brusius-Facchin, Marion Deon, Gabriel Eduardo Santiago Civallero, Roberto Giugliani
{"title":"巴西IEM诊断网络:LSD巴西网络10年活动报告,帮助巴西和拉丁美洲识别溶酶体贮积病患者。","authors":"Franciele Barbosa Trapp, Mariluce Riegel, Kristiane Michelin-Tirelli, Layzon Antonio Lemos da Silva, Gabrielle Dineck Iop, Fernanda Bender-Pasetto, Fernanda Medeiros Sebastião, Inamara da Silva Moraes, Franciele Fátima Lopes, Osmar Rachor Toledo Dos Santos, Fernanda da Rosa Pereira, Laysla Pedelhes Silva, Andrius Anselmi Luiz, Cleiton Amaral da Silva, Karen Daniela Piccoli Lucas, Alice Brinckmann Oliveira Netto, Carolina Serpa Brasil, Sandra Leistner, Ana Carolina Brusius-Facchin, Marion Deon, Gabriel Eduardo Santiago Civallero, Roberto Giugliani","doi":"10.1007/s12687-025-00805-3","DOIUrl":null,"url":null,"abstract":"<p><p>To contribute to the diagnosis of genetic metabolic diseases in Brazil, an innovative model of networks has been set up, aiming to provide information and testing of high-risk patients, free of charge, to doctors and services from Brazil and other countries. Particularly, our group set up the MPS (mucopolysaccharidoses), IEM (inborn errors of metabolism) and LSD (lysosomal storage diseases) networks. This paper describes the structure, services provided, and results obtained by the LSD Brazil Network (LBN) during its first 10 years of operation.The LBN has a core group that is responsible for the operation, guidance, logistics of sample collection and transportation, as well as test reports and information materials. The diagnostic investigation was performed in several laboratories affiliated to the program, all established in Porto Alegre, Brazil. The first ten years of operation of the LBN (2013-2022) were reviewed in detail. Over 10 years, 26,489 patients were investigated, and 27 LSDs were confirmed in 1,320 patients (4.98%). Comparing the first period of 5 years with the second one, there was a decrease in the number of requests, probably reflecting the impact of the COVID-19 pandemic. Interestingly, the same comparison indicated that there was an increase of 46% in the rate of diagnosis, possibly due to the improved selection of suspected cases. The model developed by the LBN, aiming to increase awareness and to remove barriers to diagnostic tests, suggests that the implementation of reference networks seems to be a suitable alternative to foster the diagnosis of LSDs, especially in developing countries, where resources and facilities are scarce.</p>","PeriodicalId":46965,"journal":{"name":"Journal of Community Genetics","volume":" ","pages":""},"PeriodicalIF":1.5000,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Diagnostic networks for IEM in Brazil: report on 10 years of activity of the LSD Brazil Network, helping Brazil and Latin America to identify patients with lysosomal storage diseases.\",\"authors\":\"Franciele Barbosa Trapp, Mariluce Riegel, Kristiane Michelin-Tirelli, Layzon Antonio Lemos da Silva, Gabrielle Dineck Iop, Fernanda Bender-Pasetto, Fernanda Medeiros Sebastião, Inamara da Silva Moraes, Franciele Fátima Lopes, Osmar Rachor Toledo Dos Santos, Fernanda da Rosa Pereira, Laysla Pedelhes Silva, Andrius Anselmi Luiz, Cleiton Amaral da Silva, Karen Daniela Piccoli Lucas, Alice Brinckmann Oliveira Netto, Carolina Serpa Brasil, Sandra Leistner, Ana Carolina Brusius-Facchin, Marion Deon, Gabriel Eduardo Santiago Civallero, Roberto Giugliani\",\"doi\":\"10.1007/s12687-025-00805-3\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>To contribute to the diagnosis of genetic metabolic diseases in Brazil, an innovative model of networks has been set up, aiming to provide information and testing of high-risk patients, free of charge, to doctors and services from Brazil and other countries. Particularly, our group set up the MPS (mucopolysaccharidoses), IEM (inborn errors of metabolism) and LSD (lysosomal storage diseases) networks. This paper describes the structure, services provided, and results obtained by the LSD Brazil Network (LBN) during its first 10 years of operation.The LBN has a core group that is responsible for the operation, guidance, logistics of sample collection and transportation, as well as test reports and information materials. The diagnostic investigation was performed in several laboratories affiliated to the program, all established in Porto Alegre, Brazil. The first ten years of operation of the LBN (2013-2022) were reviewed in detail. Over 10 years, 26,489 patients were investigated, and 27 LSDs were confirmed in 1,320 patients (4.98%). Comparing the first period of 5 years with the second one, there was a decrease in the number of requests, probably reflecting the impact of the COVID-19 pandemic. Interestingly, the same comparison indicated that there was an increase of 46% in the rate of diagnosis, possibly due to the improved selection of suspected cases. The model developed by the LBN, aiming to increase awareness and to remove barriers to diagnostic tests, suggests that the implementation of reference networks seems to be a suitable alternative to foster the diagnosis of LSDs, especially in developing countries, where resources and facilities are scarce.</p>\",\"PeriodicalId\":46965,\"journal\":{\"name\":\"Journal of Community Genetics\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":1.5000,\"publicationDate\":\"2025-06-02\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Community Genetics\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s12687-025-00805-3\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Community Genetics","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s12687-025-00805-3","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
Diagnostic networks for IEM in Brazil: report on 10 years of activity of the LSD Brazil Network, helping Brazil and Latin America to identify patients with lysosomal storage diseases.
To contribute to the diagnosis of genetic metabolic diseases in Brazil, an innovative model of networks has been set up, aiming to provide information and testing of high-risk patients, free of charge, to doctors and services from Brazil and other countries. Particularly, our group set up the MPS (mucopolysaccharidoses), IEM (inborn errors of metabolism) and LSD (lysosomal storage diseases) networks. This paper describes the structure, services provided, and results obtained by the LSD Brazil Network (LBN) during its first 10 years of operation.The LBN has a core group that is responsible for the operation, guidance, logistics of sample collection and transportation, as well as test reports and information materials. The diagnostic investigation was performed in several laboratories affiliated to the program, all established in Porto Alegre, Brazil. The first ten years of operation of the LBN (2013-2022) were reviewed in detail. Over 10 years, 26,489 patients were investigated, and 27 LSDs were confirmed in 1,320 patients (4.98%). Comparing the first period of 5 years with the second one, there was a decrease in the number of requests, probably reflecting the impact of the COVID-19 pandemic. Interestingly, the same comparison indicated that there was an increase of 46% in the rate of diagnosis, possibly due to the improved selection of suspected cases. The model developed by the LBN, aiming to increase awareness and to remove barriers to diagnostic tests, suggests that the implementation of reference networks seems to be a suitable alternative to foster the diagnosis of LSDs, especially in developing countries, where resources and facilities are scarce.
期刊介绍:
The Journal of Community Genetics is an international forum for research in the ever-expanding field of community genetics, the art and science of applying medical genetics to human communities for the benefit of their individuals.
Community genetics comprises all activities which identify persons at increased genetic risk and has an interest in assessing this risk, in order to enable those at risk to make informed decisions. Community genetics services thus encompass such activities as genetic screening, registration of genetic conditions in the population, routine preconceptional and prenatal genetic consultations, public education on genetic issues, and public debate on related ethical issues.
The Journal of Community Genetics has a multidisciplinary scope. It covers medical genetics, epidemiology, genetics in primary care, public health aspects of genetics, and ethical, legal, social and economic issues. Its intention is to serve as a forum for community genetics worldwide, with a focus on low- and middle-income countries.
The journal features original research papers, reviews, short communications, program reports, news, and correspondence. Program reports describe illustrative projects in the field of community genetics, e.g., design and progress of an educational program or the protocol and achievement of a gene bank. Case reports describing individual patients are not accepted.