Daniela Gerent Petry Piotto, K. Kozu, N. E. Aikawa, Pedro Lopes Carneiro, M. Katsicas, S K Oliveira, Taciana de Albuquerque Pedrosa Fernandes, Claudia Saad Magalhães, Ana Luiza Garcia Cunha, B. Bica, C. R. Rabelo Júnior, Cristina Battagliotti, E. N. Matos, F P Santos, F. Sztajnbok, Liliana Bezrodnik, M. Bandeira, M. Rodrigues, Pablo García Munittis, Simone Appenzeller, Teresa C. Robazzi, G. Clemente, C. A. Silva, M. Terreri
{"title":"自身炎症性疾病:根据年龄和性别进行的拉丁美洲多中心研究","authors":"Daniela Gerent Petry Piotto, K. Kozu, N. E. Aikawa, Pedro Lopes Carneiro, M. Katsicas, S K Oliveira, Taciana de Albuquerque Pedrosa Fernandes, Claudia Saad Magalhães, Ana Luiza Garcia Cunha, B. Bica, C. R. Rabelo Júnior, Cristina Battagliotti, E. N. Matos, F P Santos, F. Sztajnbok, Liliana Bezrodnik, M. Bandeira, M. Rodrigues, Pablo García Munittis, Simone Appenzeller, Teresa C. Robazzi, G. Clemente, C. A. Silva, M. Terreri","doi":"10.1590/1984-0462/2024/42/2022184","DOIUrl":null,"url":null,"abstract":"ABSTRACT Objective: To evaluate autoinflammatory diseases (AID) according to age at diagnosis and sex, and response to therapy in a large population. Methods: This is a cross-sectional observational study of a Latin American registry using a designed web system for data storage, collected between 2015 and 2018. Any altered findings during follow-up were recorded. The forms were translated into Portuguese and Spanish, including demographic, clinical, laboratory, genetic and treatment characteristics. Results: We included 152 patients, 51.3% male and 75% Caucasian. The median age at disease onset was 2.1 years (0–15.6 years) and median age at diagnosis 6.9 years (0–21.9 years); 111 (73%) were children (0–9 years old), and 41 (27%) were adolescents and young adults (AYA) (10–21 years old). Periodic fever, aphthous stomatitis, pharyngitis, and adenitis syndrome (PFAPA) occurred in 46/152 (30%), chronic non-bacterial osteomyelitis (CNO) in 32/152 (21%), and familial Mediterranean fever (FMF) in 24/152 (15.7%). PFAPA was significantly higher in young children than in AYA (38.7% vs. 7.3%, p<0.001), while CNO were lower (13.5% vs. 41.5%, p<0.001). The frequency of females was significantly higher in CNO (28.4% vs. 14.1%, p=0.031) and lower in FMF (8.1% vs. 23.1%, p=0.011). The most used drugs were glucocorticoids, non-steroidal anti-inflammatory drugs (NSAID), and colchicine. Glucocorticoids and colchicine treatment were used in all AID with good to moderate response. However, cryopyrin-associated periodic syndromes (CAPS) seemed unresponsive to glucocorticoids. NSAIDs and methotrexate were the main medications used to treat CNO. Conclusions: Differences among AID patients were observed in the LA population regarding sex and age at disease diagnosis.","PeriodicalId":21311,"journal":{"name":"Revista Paulista De Pediatria","volume":null,"pages":null},"PeriodicalIF":1.4000,"publicationDate":"2023-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Autoinflammatory diseases: a Latin American multicenter study according to age and sex\",\"authors\":\"Daniela Gerent Petry Piotto, K. Kozu, N. E. Aikawa, Pedro Lopes Carneiro, M. Katsicas, S K Oliveira, Taciana de Albuquerque Pedrosa Fernandes, Claudia Saad Magalhães, Ana Luiza Garcia Cunha, B. Bica, C. R. Rabelo Júnior, Cristina Battagliotti, E. N. Matos, F P Santos, F. Sztajnbok, Liliana Bezrodnik, M. Bandeira, M. Rodrigues, Pablo García Munittis, Simone Appenzeller, Teresa C. Robazzi, G. Clemente, C. A. Silva, M. Terreri\",\"doi\":\"10.1590/1984-0462/2024/42/2022184\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"ABSTRACT Objective: To evaluate autoinflammatory diseases (AID) according to age at diagnosis and sex, and response to therapy in a large population. Methods: This is a cross-sectional observational study of a Latin American registry using a designed web system for data storage, collected between 2015 and 2018. Any altered findings during follow-up were recorded. The forms were translated into Portuguese and Spanish, including demographic, clinical, laboratory, genetic and treatment characteristics. Results: We included 152 patients, 51.3% male and 75% Caucasian. The median age at disease onset was 2.1 years (0–15.6 years) and median age at diagnosis 6.9 years (0–21.9 years); 111 (73%) were children (0–9 years old), and 41 (27%) were adolescents and young adults (AYA) (10–21 years old). Periodic fever, aphthous stomatitis, pharyngitis, and adenitis syndrome (PFAPA) occurred in 46/152 (30%), chronic non-bacterial osteomyelitis (CNO) in 32/152 (21%), and familial Mediterranean fever (FMF) in 24/152 (15.7%). PFAPA was significantly higher in young children than in AYA (38.7% vs. 7.3%, p<0.001), while CNO were lower (13.5% vs. 41.5%, p<0.001). The frequency of females was significantly higher in CNO (28.4% vs. 14.1%, p=0.031) and lower in FMF (8.1% vs. 23.1%, p=0.011). The most used drugs were glucocorticoids, non-steroidal anti-inflammatory drugs (NSAID), and colchicine. Glucocorticoids and colchicine treatment were used in all AID with good to moderate response. However, cryopyrin-associated periodic syndromes (CAPS) seemed unresponsive to glucocorticoids. NSAIDs and methotrexate were the main medications used to treat CNO. Conclusions: Differences among AID patients were observed in the LA population regarding sex and age at disease diagnosis.\",\"PeriodicalId\":21311,\"journal\":{\"name\":\"Revista Paulista De Pediatria\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.4000,\"publicationDate\":\"2023-10-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Revista Paulista De Pediatria\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1590/1984-0462/2024/42/2022184\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Revista Paulista De Pediatria","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1590/1984-0462/2024/42/2022184","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PEDIATRICS","Score":null,"Total":0}
Autoinflammatory diseases: a Latin American multicenter study according to age and sex
ABSTRACT Objective: To evaluate autoinflammatory diseases (AID) according to age at diagnosis and sex, and response to therapy in a large population. Methods: This is a cross-sectional observational study of a Latin American registry using a designed web system for data storage, collected between 2015 and 2018. Any altered findings during follow-up were recorded. The forms were translated into Portuguese and Spanish, including demographic, clinical, laboratory, genetic and treatment characteristics. Results: We included 152 patients, 51.3% male and 75% Caucasian. The median age at disease onset was 2.1 years (0–15.6 years) and median age at diagnosis 6.9 years (0–21.9 years); 111 (73%) were children (0–9 years old), and 41 (27%) were adolescents and young adults (AYA) (10–21 years old). Periodic fever, aphthous stomatitis, pharyngitis, and adenitis syndrome (PFAPA) occurred in 46/152 (30%), chronic non-bacterial osteomyelitis (CNO) in 32/152 (21%), and familial Mediterranean fever (FMF) in 24/152 (15.7%). PFAPA was significantly higher in young children than in AYA (38.7% vs. 7.3%, p<0.001), while CNO were lower (13.5% vs. 41.5%, p<0.001). The frequency of females was significantly higher in CNO (28.4% vs. 14.1%, p=0.031) and lower in FMF (8.1% vs. 23.1%, p=0.011). The most used drugs were glucocorticoids, non-steroidal anti-inflammatory drugs (NSAID), and colchicine. Glucocorticoids and colchicine treatment were used in all AID with good to moderate response. However, cryopyrin-associated periodic syndromes (CAPS) seemed unresponsive to glucocorticoids. NSAIDs and methotrexate were the main medications used to treat CNO. Conclusions: Differences among AID patients were observed in the LA population regarding sex and age at disease diagnosis.
期刊介绍:
The Revista Paulista de Pediatria publishes original contributions, case reports and review of clinical research with methodological approach in the areas of health and disease of neonates, infants, children and adolescents. The objective is to disseminate research with methodological quality on issues that comprise the health of children and adolescents. All articles are freely available online, via SciELO. Its abbreviated title is Rev. Paul. Pediatr., which should be used in bibliographies, footnotes and bibliographical references and strips.