Background: Congenital adrenal hyperplasia (CAH) is a rare condition characterised by adrenal insufficiency and a life-long risk of adrenal crises. There is a paucity of information on the epidemiology of acute adverse events in this population.
Objective: To investigate the frequency, aetiology and consequences of acute adverse events attributed to adrenal insufficiency in CAH.
Methods: A longitudinal analysis of patients with CAH in the International Congenital Adrenal Hyperplasia Registry (I-CAH registry, www.i-cah.org) which collects information on acute adverse events including sick day episodes and adrenal crises.
Results: 509 patients (n= 478, 21-OHD) from 31 centres in 16 countries and a total of 3880 visits were evaluated. 261 patients (n=255, 21-OHD) had one or more sick day episodes (684 visits); of these, 215 (82%) were less than 18 years of age. 1034 sick day episodes were recorded in total, with 920 (89%) episodes recorded in those less than 18 years of age. The overall median number of sick day episodes for all centres per patient year was 3.0 for children (IQR 1.7-4.7) and 3.9 for adults (IQR 1.8-10.2) (P=0.26). The median duration of sick day episodes was 3 days (IQR 2.0-5.0) and 2 days (IQR 1.0-3.0) in children and adults respectively (P<0.05). During childhood, younger age and low hydrocortisone dose (mg/m2/day) were associated with a greater number of sick day episodes (P<0.01). Female sex was associated with higher rates of admission amongst both children and adults (P<0.01). Infectious illness was the most frequent event causing illness episodes and adrenal crises in both children (66%) and adults (23%). An adrenal crisis was reported in 37 (4%, 37/920) and 34 (30%, 34/114) sick day episodes amongst children and adults, respectively (P<0.05) and all adults required hospital admission.
Conclusions: The real world data within the I-CAH registry are a valuable resource for studying a core clinical outcome that can be used as a benchmark for improving clinical care. Further work needs to be undertaken to understand the determinants of the observed variations in the occurrence of sick day episodes.
19 - 21 Sep 2019
European Society for Paediatric Endocrinology