Endocrine Abstracts

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...

Background: In children, Cushing’s disease (CD) presents with a combination of weight gain and slowed linear growth. First-line pituitary surgery is the treatment of choice for most patients. In paediatric patients, the transsphenoidal surgical success rate is 60%–98% when performed by an expert pituitary surgeon. There is a need for additional pharmacological interventions to control hypercortisolaemia, which are currently limited, in children and adolescents. In ph...

Introduction: LNSC has shown high sensitivity and specificity for the initial diagnosis of CD and detection of disease recurrence; however, the use of LNSC to monitor medical treatment of CD is not well established. The results of an exploratory analysis evaluating changes in LNSC in CD patients receiving long-acting pasireotide during a Phase III study (CSOM230G2304; Lacroix et al. Lancet Diabetes Endocrinol 2018) are reported here.Methods: Pat...

Introduction: Long-acting pasireotide reduced urinary free cortisol (UFC) in most patients with Cushing’s disease (CD) during a large Phase III study (Lacroix et al. Lancet Diabetes Endocrinol 2018). The analyses presented here explored the impact of baseline characteristics on response to long-acting pasireotide.Methods: 150 patients with persistent, recurrent or de novo CD and mean UFC (mUFC; from three 24-hour samples collected ...

IntroductionOsilodrostat, a potent oral 11β-hydroxylase inhibitor, normalized mean urinary free cortisol (mUFC) in most patients with CD during the 48-week (W) core phase of a Phase III study (LINC3: NCT02180217). We present efficacy and safety results following an extension to LINC3.MethodsCD patients with mUFC > 1.5× upper limit of normal (ULN) received osilodrostat during the core. Patients b...

Introduction: The 12-month results of a multicentre, double-blind, Phase III study showing the efficacy and safety of a monthly, long-acting formulation of pasireotide in Cushing’s disease (CD) patients have been reported previously (Lacroix et al. Lancet Diabetes Endocrinol 2018). The results of the extension phase of this study are reported here.Methods: Patients (n=150) with persistent/recurrent or de novo CD and mean u...

IntroductionOsilodrostat, a potent, oral 11β-hydroxylase inhibitor, normalized mean urinary free cortisol (mUFC) in most patients with CD during a Phase III, randomized-withdrawal study. We now report findings from a Phase III study of osilodrostat in patients with CD that featured an initial double-blind, randomized, placebo-controlled period (LINC 4; NCT02697734).MethodsAdults with CD (mUFC > 1.3 &...