Xanthippi Tseretopoulou, Salma R Ali, Jillian Bryce, Nadia Amin, Navoda Atapattu, Tania A S S Bachega, Federico Baronio, Rita Ortolano, Niels H Birkebaek, Walter Bonfig, Martine Cools, Justin H Davies, Tessy Thomas, Liat de Vries, Heba Elsedfy, Nermine H Amr, Christa E Flueck, Evgenia Globa, Tulay Guran, Zehra Yavas-Abali, Ayla Guven, Sabine E Hannema, Violeta Iotova, Daniel Konrad, Nina Lenherr-Taube, Nils P Krone, Sofia Leka-Emiri, Elpis Vlachopapadopoulou, Corina Lichiardopol, Otilia Marginean, Renata Markosyan, Uta Neumann, Marek Niedziela, Magdalena Banaszak-Ziemska, Franziska Phan-Hug, Sukran Poyrazoglu, Ursina Probst-Scheidegger, Tabitha Randell, Gianni Russo, Mariacarolina Salerno, Sumudu Seneviratne, Margarett Shnorhavorian, Ajay Thankamony, Rieko Tadokoro-Curraro, Erica van den Akker, Judith van Eck, Ana Vieites, Malgorzata Wasniewska, S Faisal Ahmed
{"title":"Temporal Trends in Acute Adrenal Insufficiency Events in Children With Congenital Adrenal Hyperplasia During 2019-2022.","authors":"Xanthippi Tseretopoulou, Salma R Ali, Jillian Bryce, Nadia Amin, Navoda Atapattu, Tania A S S Bachega, Federico Baronio, Rita Ortolano, Niels H Birkebaek, Walter Bonfig, Martine Cools, Justin H Davies, Tessy Thomas, Liat de Vries, Heba Elsedfy, Nermine H Amr, Christa E Flueck, Evgenia Globa, Tulay Guran, Zehra Yavas-Abali, Ayla Guven, Sabine E Hannema, Violeta Iotova, Daniel Konrad, Nina Lenherr-Taube, Nils P Krone, Sofia Leka-Emiri, Elpis Vlachopapadopoulou, Corina Lichiardopol, Otilia Marginean, Renata Markosyan, Uta Neumann, Marek Niedziela, Magdalena Banaszak-Ziemska, Franziska Phan-Hug, Sukran Poyrazoglu, Ursina Probst-Scheidegger, Tabitha Randell, Gianni Russo, Mariacarolina Salerno, Sumudu Seneviratne, Margarett Shnorhavorian, Ajay Thankamony, Rieko Tadokoro-Curraro, Erica van den Akker, Judith van Eck, Ana Vieites, Malgorzata Wasniewska, S Faisal Ahmed","doi":"10.1210/jendso/bvae145","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>It is unclear whether targeted monitoring of acute adrenal insufficiency (AI) related adverse events (AE) such as sick day episodes (SDEs) and hospitalization rate in congenital adrenal hyperplasia (CAH) is associated with a change in the occurrence of these events.</p><p><strong>Aim: </strong>Study temporal trends of AI related AE in the I-CAH Registry.</p><p><strong>Methods: </strong>In 2022, data on the occurrence of AI-related AE in children aged <18 years with 21-hydroxylase deficiency CAH were compared to data collected in 2019.</p><p><strong>Results: </strong>In 2022, a total of 513 children from 38 centers in 21 countries with a median of 8 children (range 1-58) per center had 2470 visits evaluated over a 3-year period (2019-2022). The median SDE per patient year in 2022 was 0 (0-2.5) compared to 0.3 (0-6) in 2019 (<i>P</i> = .01). Despite adjustment for age, CAH phenotype and duration of study period, a difference in SDE rate was still apparent between the 2 cohorts. Of the 38 centers in the 2022 cohort, 21 had also participated in 2019 and a reduction in SDE rate was noted in 13 (62%), an increase was noted in 3 (14%), and in 5 (24%) the rate remained the same. Of the 474 SDEs reported in the 2022 cohort, 103 (22%) led to hospitalization compared to 299 of 1099 SDEs (27%) in the 2019 cohort (<i>P</i> = .02).</p><p><strong>Conclusion: </strong>The I-CAH Registry can be used for targeted monitoring of important clinical benchmarks in CAH. However, changes in reported benchmarks need careful interpretation and longer-term monitoring.</p>","PeriodicalId":17334,"journal":{"name":"Journal of the Endocrine Society","volume":"8 10","pages":"bvae145"},"PeriodicalIF":3.0000,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11387114/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of the Endocrine Society","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1210/jendso/bvae145","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/8/27 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
引用次数: 0
Abstract
Background: It is unclear whether targeted monitoring of acute adrenal insufficiency (AI) related adverse events (AE) such as sick day episodes (SDEs) and hospitalization rate in congenital adrenal hyperplasia (CAH) is associated with a change in the occurrence of these events.
Aim: Study temporal trends of AI related AE in the I-CAH Registry.
Methods: In 2022, data on the occurrence of AI-related AE in children aged <18 years with 21-hydroxylase deficiency CAH were compared to data collected in 2019.
Results: In 2022, a total of 513 children from 38 centers in 21 countries with a median of 8 children (range 1-58) per center had 2470 visits evaluated over a 3-year period (2019-2022). The median SDE per patient year in 2022 was 0 (0-2.5) compared to 0.3 (0-6) in 2019 (P = .01). Despite adjustment for age, CAH phenotype and duration of study period, a difference in SDE rate was still apparent between the 2 cohorts. Of the 38 centers in the 2022 cohort, 21 had also participated in 2019 and a reduction in SDE rate was noted in 13 (62%), an increase was noted in 3 (14%), and in 5 (24%) the rate remained the same. Of the 474 SDEs reported in the 2022 cohort, 103 (22%) led to hospitalization compared to 299 of 1099 SDEs (27%) in the 2019 cohort (P = .02).
Conclusion: The I-CAH Registry can be used for targeted monitoring of important clinical benchmarks in CAH. However, changes in reported benchmarks need careful interpretation and longer-term monitoring.
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