Endocrine Abstracts

Introduction: In patients with β thalassemia major (TM) The interplay between liver siderosis and hepatitis C virus (HCV) infection may facilitates the progression to insulin resistance (IR) and diabetes mellitus (DM).Objectives: Many TM patients are infected with either HCV. Therefore, we aimed to explore if there is any association between DM and HCV-RNA positivity with different genotypes.Patients and methods: 148 TM patien...

GHD in adults (AGHD) is a clinical syndrome associated with lack of positive well-being, depressed mood, feelings of social isolation, decreased energy, alterations in body composition with reduced bone and muscle mass, diminished exercise performance, and cardiac capacity. These manifestations are also common in adults with beta-thalassemia major (BTM). Performing provocative testing in all patients is cumbersome and expensive. Many studies suggested that IGF1 may be used for...

Background: TransCon hGH is a long-acting prodrug of recombinant human GH (hGH) that releases into the blood compartment fully active unmodified hGH. TransCon hGH was shown in two Phase 1 studies and a Phase 2 study in AGHD to be safe and well tolerated and inducing an IGF-I pharmacodynamic response within the normal range throughout the dosing period. This interim analysis consists of 25 patients completing all 6 months of treatment.Objectives: The obje...

Background: Congenital adrenal hyperplasia (CAH) is a rare condition that is associated with life long risk of adrenal crisis. Management of CAH demands a fine balance between excess glucocorticoid leading to adverse effects and too little glucocorticoid risking adrenal crises. Frequent occurrence of sick day episodes warrants dose adjustment and education regarding adrenal crisis. In a condition such as CAH it is difficult to collect sufficient data from small cohorts at a si...

Background: Congenital Adrenal Hyperplasia (CAH) requires exogenous steroid replacement and can be monitored with 17-OH Progesterone and Androstenedione. We reviewed real world data to evaluate these markers in relation to hydrocortisone dose in patients treated in 21 centres throughout 14 countries.Method: Retrospective cohort study using pseudonymised data from patients with 21α-Hydroxylase Deficiency recorded in the International Congenital Adren...

Background: Testicular Adrenal Rest Tumors (TART) in patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD) or 11-hydroxylase deficiency (11OHD) are benign lesions causing testicular damage and infertility. We hypothesize that high ACTH exposure due to poor hormonal control during early life is promoting development of TART later in life.Objective: This study aims to examine the relation between early CAH diagnosis and cons...

Introduction: There is no unified approach in clinical practice regarding the medical management of congenital adrenal hyperplasia (CAH), despite existent international guidance. We aimed to explore geographical and temporal variations in the treatment with glucocorticoids and mineralocorticoids of patients with CAH.Methods: We collected data recorded by 33 centres from 16 countries in the I-CAH Registry. We analysed patient visits between 1982 and 2018,...

Background: In 2019, the International CAH Registry (I-CAH) performed a benchmarking exercise of acute adrenal insufficiency related adverse events including adrenal crises (AC) and sick day episodes (SDE).Methods: In 2022, I-CAH data on children aged <18 years at first visit with 21-hydroxylase deficiency CAH from 35 centres in 19 countries were analysed to examine the current occurrence of SDE and AC and compared t...

Background: The reported occurrence and management of acute adrenal insufficiency–related adverse events in children vary widely between centres and may depend on available resources.Methods: Real world data from the I-CAH Registry from 44 centres [32 from high income (HIC) and 12 from low/middle income (LMIC) countries] and a total number of 607 children were linked to the results of a health care survey of local r...

Background: The reported occurrence and management of acute adrenal insufficiency–related adverse events in children vary widely between centres and may depend on available resources.Methods: Real world data from the I-CAH Registry that included a total of 2478 patient years follow-up on 607 children from 44 centres were studied and their association to the results of a health care survey of these centres that enqui...