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, M. rta
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
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.
UR - https://www.scopus.com/pages/publications/85099073104
UR - https://www.ncbi.nlm.nih.gov/pubmed/32995889
U2 - 10.1210/clinem/dgaa694
DO - 10.1210/clinem/dgaa694
M3 - Article
C2 - 32995889
SN - 0021-972X
VL - 106
SP - E192-E203
JO - Journal of clinical endocrinology and metabolism
JF - Journal of clinical endocrinology and metabolism
IS - 1
ER -