Endocrine Abstracts

Introduction: Cushing’s disease (CD) is a severe disease, associated with an increased rate of comorbidities and mortality. Remission rate after surgery of pituitary tumor, is around 78%. Relapse occurs in 13% of patients within 10-years after surgery. According to guidelines, patients with unfeasible or non-curative surgery, require additional treatment, including medical therapies. Metyrapone, inhibits 11ß-hydroxylase enzyme, blocking the final step of cortisol syn...

Introduction: Metyrapone blocks cortisol production by inhibiting 11ß-hydroxylation of 11-deoxycortisol, the last step of cortisol synthesis. Based on observational retrospective studies published over more than 50 years metyrapone is approved for the treatment of endogenous Cushing’s syndrome (CS) in 14 European countries. PROMPT is the first prospective study to document the safety and efficacy of metyrapone using modern assay techniques.Desi...

Introduction and rationale: Cushing’s syndrome (CS) is a rare disease with hypercortisolism caused either by ACTH excess from a pituitary or non-pituitary tumor or by an ACTH-independent primary adrenal overproduction of cortisol. It is associated with significant comorbidities potentially lethal: hypertension, diabetes, coagulopathy, cardiovascular disease, infections, and osteoporotic fractures. It is usually managed by surgery and/or medical treatment with steroidogene...

Metyrapone treatment in endogenous Cushing’s syndrome. Results from a prospective multicenter, open-label, phase III/IV study: PROMPTBackground: Metyrapone is a steroidogenesis inhibitor approved in Europe for the treatment of endogenous Cushing’s syndrome (CS) based on observational retrospective studies published over more than 50 years. We present data from the first prospective study designed to confirm metyrapone efficacy and good tolera...

BackgroundRetrospective studies led to European approval of the steroidogenesis inhibitor Metyrapone for the treatment of endogenous Cushing’s syndrome (CS). We prospectively showed good efficacy and safety of Metyrapone after 12 weeks (Wk12) treatment in the phase III/IV PROMPT study and now report results of an extension study (EXT) sponsored by HRA Pharma Rare Diseases.DesignThis was a single arm, ope...