Endocrine Abstracts

Background: Quality of life (QoL) has been variously reported as normal or impaired in congenital adrenal hyperplasia (CAH) adults. We found impaired QoL in UK CAH adults and now report the relationship between QoL, glucocorticoid treatment and health outcomes in these patients.Methods: Cross-sectional analysis of 151 CAH adults with 21-hydroxylase deficiency aged 18–69 years in whom QoL (SF-36), glucocorticoid regimen, anthropometric, and metabolic...

We have previously reported the following metabolic abnormalities were common in 203 adult patients with CAH: obesity (41%), hypercholesterolemia (46%), insulin resistance (29%), osteopenia (40%) and osteoporosis (7%) (Arlt et al. JCEM 2010 95 5110–21). The CAH patients were taking different glucocorticoid therapies at various doses (n=196): hydrocortisone (n=25 M, 26 W), prednisolone (n=21M,67W), dexamethasone (n=15M,22W) or comb...

Neuroendocrine tumors (NETs) of GI tract are a heterogeneous group of neoplasms that secrete peptides and amines. Small bowel NETs originate from enterochromaffin (EC) cells and secrete chromogranin A (CgA) and serotonin (5-HT). NETs are highly vascular, a reflection of increased tumour related angiogenesis. Adenosine, the major regulator of angiogenesis, is released by enhanced degradation of ATP, during cellular stress, damage and hypoxia.Aims: Aims of...

Background: The Congenital adrenal Hyperplasia (CAH) Adult Study Executive (CaHASE) identified poor metabolic outcomes and reduced quality of life in CAH. CaHASE2 was recently established to examine the current status of CAH care. We surveyed clinical practice in the UK and Ireland, and awareness and use of the International CAH (I-CAH) Registry.Methods: We undertook an anonymised online survey targeting clinicians provi...

Adrenal crisis (AC) is a life-threatening episode of adrenal insufficiency (AI) resulting from impairment of glucocorticoid secretion by the adrenal cortex. Approximately 1 in 200 patients die from AC annually.1 AC is often precipitated by intercurrent illness, injury or surgery and can be prevented by increasing oral corticosteroid doses (‘stress dosing’) during illness. Parenteral hydrocortisone may be necessary if oral therapy cannot be absorbed or inef...

Introduction: First-line treatment for congenital adrenal hyperplasia (CAH) is hydrocortisone1. When adequate control is not achieved, prednisolone (or its prodrug prednisone) are often used. However, there has been no formal comparison of disease control in CAH comparing prednis(ol)one vs hydrocortisone and patients are often on a glucocorticoid dose that exceeds the guideline recommended dose of hydrocortisone (≤25 mg/day)1,2. We report an interim...

Introduction: The effects of maternal suboptimal gestational thyroid function (SGTF) on age 3 offspring’s cognitive function were investigated in the Controlled Antenatal Thyroid Screening (CATS) randomised trial, comparing SGTF mothers who received (SGTF-T), or didn’t (SGTF-U), levothyroxine during pregnancy. The analysis was repeated at age 9 in the CATS-II follow-up study, also including children of mothers with normal gestational thyroid function (NGTF). Here we ...

Objectives: The Controlled Antenatal Thyroid Screening (CATS) study I was a randomised trial investigating the effects of levothyroxine treatment for suboptimal gestational thyroid function (SGTF), evaluating mothers with normal gestational thyroid function (NGTF), SGTF who received (SGTF-T), or didn’t (SGTF-U), levothyroxine during pregnancy. The present follow-up study (CATS II) reports the long-term effects of SGTF and levothyroxine treatment on anthropometric and card...

ATL1103 is a second generation antisense 20mer intended to inhibit expression of the GH receptor (GHR) gene. Phosphorothioate and 2′-O-methoxyethyl modifications to nucleotides increase its plasma half-life and affinity for the target RNA to allow post-hybridization RNaseH degradation. We previously reported a phase 2, randomised, open-label, parallel group study of ATL1103 in 26 patients with acromegaly which demonstrated a fall in serum IGF-I of 26% with 200 mg twice w...

ATL1103 is a second generation antisense oligomer directed at the GH receptor. It is a 20mer with a phosphorothioate backbone and 2′-O-methoxyethyl modifications of the five nucleotides at either end intended to increase its plasma half-life and affinity for the target RNA to allow post-hybridization RNaseH degradation. We report a phase 2 randomised, open-label, parallel group study of subcutaneously administered ATL1103 in patients with active acromegaly. Appr...