Endocrine Abstracts

Background: Management of CAH involves replacing cortisol deficiency and reducing raised adrenal androgens, however the supraphysiological glucocorticoid doses often required to treat hyperandrogenism are associated with poor long-term health outcomes. Modified-release hydrocortisone (MRHC) capsules, Efmody, replicate cortisol diurnal rhythm and improve control of CAH compared to standard glucocorticoid therapy. Here we report changes in glucocorticoid daily dose and 9am 17-hy...

Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most common cause of chronic liver disease and is the leading cause of liver-related morbidity and mortality. MASLD is rapidly becoming a major aetiology for the development of hepatocellular carcinoma (HCC), the most common liver cancer. As the incidence of MASLD continues to rise, it is crucial to understand the molecular alterations that lead to HCC. Previous studies have revealed a significant alterati...

Glioblastoma (GBM) is the most prevalent and most lethal primary brain endocrine-related cancer (ERC) in adults which, in addition to the late-stage diagnosis and the lack of effective novel therapies, results in the low quality of life of patients and the poor prognosis, with a median survival from 10 to 14 months after diagnosis. Accordingly, the identification of novel molecular biomarkers of diagnosis and/or prognosis, as well as therapeutic targets becomes crucial to comb...

Background: Patients with congenital adrenal hyperplasia (CAH) due to classic 21-hydroxylase deficiency have poor health outcomes. We compared disease control in CAH adults treated with modified release hydrocortisone (MRHC, Chronocort®, Diurnal Ltd) versus standard glucocorticoid (GC).Methods: 6-month, Phase 3 study in 122 patients randomised to either MRHC twice daily or standard GC followed by safety extension study on MRHC. Patients had 24-hr 17...

Background: The therapeutic goal in CAH is androgen control on the lowest achievable glucocorticoid dose, preferably an adrenal replacement dose (15-25 mg hydrocortisone a day)1. However, the glucocorticoid dose required to control androgens frequently exceeds that required for adrenal replacement2. Modified-release hydrocortisone (MRHC) capsules, (Efmody, Diurnal Ltd, Cardiff, UK), replicate cortisol diurnal rhythm and improve CAH control compared to sta...

Background: Patients with salt-wasting congenital adrenal hyperplasia (CAH) due to classic 21-hydroxylase deficiency require glucocorticoid (GC) and mineralocorticoid (MC) replacement therapy. Recently, it was shown that twice daily modified-release hydrocortisone hard capsules (MRHC, Efmody®, Diurnal Ltd) improved control of CAH with most patients showing good disease control versus standard GC therapy. However, no data has been reported on the renin-angiotens...

Background: Fertility in CAH women is impaired: 0.25 live births vs 1.8 in the UK population and 45% have irregular menses vs 13.6% in healthy women1. Male fertility is also impaired in CAH with oligospermia reported in 48%2. Treatment of infertility usually involves increasing the glucocorticoid dose to normalise adrenal androgens and progesterone to facilitate ovulation and implantation, respectively. Modified-release hydrocortisone (MRHC) capsules, (Ef...

Introduction: Hypothyroidism has been associated with oxidative stress. Urinary excretion of 8-oxo-7,8-dihydroguanosine (8-oxoGuo) and 8-oxo-7,8-dihydro-2’-deoxyguanosine (8-oxodG), respectively, represent whole-body RNA and DNA oxidation. While these biomarkers have been associated with increased morbidity and mortality in various diseases, they have been only sparsely explored in patients with thyroid disorders.Method: In 45 women with newly diagn...

Background: Modified-release hydrocortisone (MRHC) capsules (Efmody, Diurnal Ltd, Cardiff, UK), have been developed to replicate the cortisol diurnal rhythm and shown to improve CAH disease control1. We have examined relative bioavailability of MRHC and disease control of congenital adrenal hyperplasia (CAH) patients switched from standard therapy to MRHC.Methods: An open label, randomised, 2 period, crossover study comparing the relative bioavailability...

The incidence of Neuroendocrine Neoplasm (NEN) and related carcinoid syndrome (CS) increased markedly in recent decades, and women appear to be more at risk than men. As per other tumors, gender may be relevant in influencing the clinical and prognostic characteristics. Unfortunately, there are no studies designed to answer this question in the literature. The present multicentric study was designed to evaluated gender differences, if any, in clinical presentation and outcomes...