Endocrine Abstracts

Introduction: Patients with adrenal insufficiency (AI) (primary (PAI), secondary to pituitary disease (PIT) and congenital adrenal hyperplasia (CAH)) have reduced life expectancy with reported standardized mortality ratios of ~2:1 but given the rarity of AI, the underlying explanation remains largely unknown.Objective: To evaluate patient characteristics, prevalence of concomitant conditions and hospitalization incidence in patients with AI compared to a...

Background: Twice-daily formulation of pasireotide, a pituitary-directed therapy, is approved for treatment of Cushing’s disease. Here we present data from a phase III study designed to evaluate the more convenient once-monthly long-acting release (LAR) formulation of pasireotide (approved for acromegaly) in patients with Cushing’s disease.Methods: Patients with persistent, recurrent, or de novo Cushing’s disease (not candidates for surger...

Introduction: The clinical outcome of patients with adrenal insufficiency (AI) has been shown to be less favorable than previously thought. Clinical studies have shown increased mortality, reduced cardiovascular and skeletal health and compromised quality of life, but the impact of this upon healthcare burden is unknown. This research utilised real-world evidence to compare comorbidities, healthcare utilization and expenditures in patients with AI.Method...

Recombinant human growth hormone (GH) is not indicated for use during pregnancy and in women of childbearing potential not using contraception. Nonetheless, in clinical practice, some women taking GH replacement conceive during treatment and many continue GH during their pregnancy. Here we report data on GH-treated women enrolled in two complementary, international, non-interventional registry studies, NordiNet IOS (NCT00960128; 2006–2016) and ANSWER (NCT01009905; 2002&#...

Background: Improving physical manifestations of hypercortisolism is an important treatment goal for patients with Cushing’s disease (CD). In the Phase III LINC 3 study (NCT02180217), osilodrostat therapy, a potent oral 11β-hydroxylase inhibitor, rapidly normalised mean urinary free cortisol (mUFC) in most patients with CD and sustained control of mUFC over a median treatment period of 130 weeks (W). Here we describe concomitant improvements in physical manifestation...

Introduction: Osilodrostat decreases cortisol production by inhibiting 11β-hydroxylase, increasing adrenal hormones above the blockade. Here, we describe these effects of osilodrostat and associated adverse events (AEs). The efficacy and safety of osilodrostat in patients with Cushing’s disease (CD) were confirmed in the published Phase III, prospective LINC 3 study (NCT02180217).Methods: 137 patients with CD (mUFC >1.5x upper limit of norm...

Background: 24-h mean urinary free cortisol (mUFC) and late-night salivary cortisol (LNSC) levels are complementary parameters recommended for screening and monitoring treatment response in patients with Cushing’s disease (CD). In the published core period of the Phase III LINC 3 study (NCT02180217), therapy with osilodrostat (potent oral 11β-hydroxylase inhibitor) produced rapid, sustained reductions in mUFC and LNSC alongside improvements in clinical signs of hyper...

Introduction: There are limited data on the effectiveness and safety of GHRT in older patients with AGHD. We compared real-world GHRT outcomes in older (aged ≥60 years) vs middle-aged (35–<60 years) adults.Methods: NordiNet® IOS (NCT00960128) and ANSWER (NCT01009905) were non-interventional studies investigating long-term effectiveness and safety of GHRT with Norditropin® (somatropin, Novo Nordisk). Safety was assessed in the ful...

Background: Following the 34-week, double-blind, placebo-controlled main phase of REAL-1 (NCT02229851), this open-label trial extension evaluated efficacy and safety of somapacitan in patients aged 23–79 years with AGHD, for an additional 52 weeks (8 weeks’ dose titration followed by 44 weeks’ fixed dose treatment; 86 weeks’ treatment in total).Methods: Patients completing the main trial entered the extension: 1) somapacitan-treated p...

Introduction: Hypertension (HTN) is a major cardiovascular (CV) risk factor and independent predictor of the increased mortality in patients with acromegaly. Surgical cure of acromegaly is associated with improvement in blood pressure (BP) levels, however little is known on the effect of pegvisomant (PEGV) treatment on HTN.Methods/design: ACROSTUDY is an open-label, international, prospective, non-interventional study monitoring the long-term safety of P...