TY - JOUR
T1 - Real-World Estimates of Adrenal Insufficiency-Related Adverse Events in Children with Congenital Adrenal Hyperplasia
AU - Ali, Salma R
AU - Bryce, Jillian
AU - Haghpanahan, Houra
AU - Lewsey, James D
AU - Tan, Li En
AU - Atapattu, Navoda
AU - Birkebaek, Niels H
AU - Blankenstein, Oliver
AU - Neumann, Uta
AU - Balsamo, Antonio
AU - Ortolano, Rita
AU - Bonfig, Walter
AU - Claahsen-van der Grinten, Hedi L
AU - Cools, Martine
AU - Costa, Eduardo Correa
AU - Darendeliler, Feyza
AU - Poyrazoglu, Sukran
AU - Elsedfy, Heba
AU - Finken, Martijn J J
AU - Fluck, Christa E
AU - Gevers, Evelien
AU - Korbonits, Márta
AU - Guaragna-Filho, Guilherme
AU - Guran, Tulay
AU - Guven, Ayla
AU - Hannema, Sabine E
AU - Higham, Claire
AU - Hughes, Ieuan A
AU - Tadokoro-Cuccaro, Rieko
AU - Thankamony, Ajay
AU - Iotova, Violeta
AU - Krone, Nils P
AU - Krone, Ruth
AU - Lichiardopol, Corina
AU - Luczay, Andrea
AU - Mendonca, Berenice B
AU - Bachega, Tania A S S
AU - Miranda, Mirela C
AU - Milenkovic, Tatjana
AU - Mohnike, Klaus
AU - Nordenstrom, Anna
AU - Einaudi, Silvia
AU - van der Kamp, Hetty
AU - Vieites, Ana
AU - de Vries, Liat
AU - Ross, Richard J M
AU - Ahmed, S Faisal
N1 - 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; Charité Universitätsmedizin Berlin; Ottovon- Guericke University, Magdeburg; Ludwig-Maximilian- University Munich; Semmelweis University, Budapest; Azienda Ospedaliera Universitaria Sant'Orsola Malpighi, Bologna; Azienda Ospedaliero Universitaria Città 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).
PY - 2021/1/1
Y1 - 2021/1/1
N2 - 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.
AB - 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.
KW - 21-hydroxylase deficiency
KW - adrenal crisis
KW - adrenal insufficiency
KW - congenital adrenal hyperplasia
KW - registry
UR - http://www.scopus.com/inward/record.url?scp=85099073104&partnerID=8YFLogxK
U2 - 10.1210/clinem/dgaa694
DO - 10.1210/clinem/dgaa694
M3 - Article
C2 - 32995889
SN - 0021-972X
VL - 106
SP - E192-E203
JO - The Journal of clinical endocrinology and metabolism
JF - The Journal of clinical endocrinology and metabolism
IS - 1
ER -