Real-World Estimates of Adrenal Insufficiency-Related Adverse Events in Children with Congenital Adrenal Hyperplasia

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DOI

  • Salma R. Ali, University of Glasgow, Royal Hospital for Children, Glasgow
  • ,
  • Jillian Bryce, Royal Hospital for Children, Glasgow
  • ,
  • Houra Haghpanahan, University of Glasgow
  • ,
  • James D. Lewsey, University of Glasgow
  • ,
  • Li En Tan, University of Glasgow
  • ,
  • Navoda Atapattu, Lady Ridgeway Hospital
  • ,
  • Niels H. Birkebaek
  • Oliver Blankenstein, Charité-Universitätsmedizin Berlin
  • ,
  • Uta Neumann, Charité-Universitätsmedizin Berlin
  • ,
  • Antonio Balsamo, University of Bologna
  • ,
  • Rita Ortolano, University of Bologna
  • ,
  • Walter Bonfig, Technical University of Munich, Klinikum Wels-Grieskirchen
  • ,
  • Hedi L. Claahsen-Van Der Grinten, Radboud University Nijmegen
  • ,
  • Martine Cools, Ghent University
  • ,
  • Eduardo Correa Costa, Universidade Federal do Rio Grande do Sul
  • ,
  • Feyza Darendeliler, Istanbul University
  • ,
  • Sukran Poyrazoglu, Istanbul University
  • ,
  • Heba Elsedfy, Ain Shams University
  • ,
  • Martijn J.J. Finken, Vrije Universiteit Amsterdam
  • ,
  • Christa E. Fluck, University of Bern
  • ,
  • Evelien Gevers, Queen Mary University of London
  • ,
  • Márta Korbonits, Queen Mary University of London
  • ,
  • Guilherme Guaragna-Filho, Universidade Federal do Rio Grande do Sul
  • ,
  • Tulay Guran, Marmara University
  • ,
  • Ayla Guven, University of Health Sciences
  • ,
  • Sabine E. Hannema, Erasmus University Rotterdam, Leiden University
  • ,
  • Claire Higham, Manchester University
  • ,
  • Ieuan A. Hughes, University of Cambridge
  • ,
  • Rieko Tadokoro-Cuccaro, University of Cambridge
  • ,
  • Ajay Thankamony, University of Cambridge
  • ,
  • Violeta Iotova, Medical University of Varna
  • ,
  • Nils P. Krone, University of Sheffield
  • ,
  • Ruth Krone, Birmingham Women's and Children's NHS Foundation Trust
  • ,
  • Corina Lichiardopol, Craiova University of Medicine and Pharmacy
  • ,
  • Andrea Luczay, Semmelweis University
  • ,
  • Berenice B. Mendonca, Universidade de Sao Paulo
  • ,
  • Tania A.S.S. Bachega, Universidade de Sao Paulo
  • ,
  • Mirela C. Miranda, Universidade de Sao Paulo
  • ,
  • Tatjana Milenkovic, Mother and Child Health Care Institute
  • ,
  • Klaus Mohnike, Otto von Guericke University
  • ,
  • Anna Nordenstrom, Karolinska Institutet
  • ,
  • Silvia Einaudi, University of Turin
  • ,
  • Hetty Van Der Kamp, Utrecht University
  • ,
  • Ana Vieites, Hospital de Ninos Ricardo Gutierrez
  • ,
  • Liat De Vries, Schneider Childrens Medical Center Israel, Tel Aviv University
  • ,
  • Richard J.M. Ross, University of Sheffield
  • ,
  • S. Faisal Ahmed, University of Glasgow, Royal Hospital for Children, Glasgow

Background: Although congenital adrenal hyperplasia (CAH) is known to be associated with adrenal crises (AC), its association with patient- or clinician-reported sick day episodes (SDE) is less clear. Methods: Data on children with classic 21-hydroxylase deficiency CAH from 34 centers in 18 countries, of which 7 were Low or Middle Income Countries (LMIC) and 11 were High Income (HIC), were collected from the International CAH Registry and analyzed to examine the clinical factors associated with SDE and AC. Results: A total of 518 children - with a median of 11 children (range 1, 53) per center - had 5388 visits evaluated over a total of 2300 patient-years. The median number of AC and SDE per patient-year per center was 0 (0, 3) and 0.4 (0.0, 13.3), respectively. Of the 1544 SDE, an AC was reported in 62 (4%), with no fatalities. Infectious illness was the most frequent precipitating event, reported in 1105 (72%) and 29 (47%) of SDE and AC, respectively. On comparing cases from LMIC and HIC, the median SDE per patient-year was 0.75 (0, 13.3) vs 0.11 (0, 12.0) (P < 0.001), respectively, and the median AC per patient-year was 0 (0, 2.2) vs 0 (0, 3.0) (P = 0.43), respectively. Conclusions: The real-world data that are collected within the I-CAH Registry show wide variability in the reported occurrence of adrenal insufficiency-related adverse events. As these data become increasingly used as a clinical benchmark in CAH care, there is a need for further research to improve and standardize the definition of SDE.

OriginalsprogEngelsk
TidsskriftJournal of Clinical Endocrinology and Metabolism
Vol/bind106
Nummer1
Sider (fra-til)E192-E203
ISSN0021-972X
DOI
StatusUdgivet - jan. 2021

Bibliografisk note

Funding Information:
This work would not be possible without the children and the parents of the children with CAH whose data have been included in the I-CAH Registry. A.T., R.C.C., and I.A.H. acknowledge the support of NIHR Cambridge Biomedical Research Centre. We would like to thank Professor Rodolfo Rey for his assistance and would also like to acknowledge the support of the following reference centers that participate in the European Reference Network for Rare Endocrine Conditions (Endo-ERN): Klinikum Wels-Grieskirchen, Center for Pediatric Endocrinology; Ghent University Hospital; MHAT Sveta Marina, Varna; Aarhus University Hospital; Charit? Universit?tsmedizin Berlin; Ottovon- Guericke University, Magdeburg; Ludwig-Maximilian- University Munich; Semmelweis University, Budapest; Azienda Ospedaliera Universitaria Sant'Orsola Malpighi, Bologna; Azienda Ospedaliero Universitaria Citt? della Salute e della Scienza di Torino; Amsterdam- University Medical Center, Leiden University Medical Center; Radboud University Nijmegen Medical Center; Erasmus Medical Center Rotterdam; University Medical Center Utrecht;, Karolinska University Hospital, Stockholm; University Hospitals Birmingham NHS Foundation Trust; NHS Greater Glasgow and Clyde Board; Barts Health NHS Foundation Trust; Central Manchester University Hospitals NHS Foundation Trust.

Publisher Copyright:
© 2020 The Author(s).

Copyright:
Copyright 2021 Elsevier B.V., All rights reserved.

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