Endocrine Abstracts

Dr Lynnette Nieman is a Senior Investigator and Chief of the Endocrinology Consultation Service at the National Institutes of Health (NIH) Clinical Research Center. She has been at the NIH since her fellowship. From 1991 to 2001 she served as the Clinical Director of intramural NICHD, overseeing clinical care of the institute’s patients and ensuring compliance with human subjects research regulations.Dr Nieman is an active clinician, having seen mor...

Objective: Ulipristal acetate or UPA (PGL4001, formerly CDB-2914) is an oral SPRM with the potential to be the first in a new treatment class for uterine fibroids. As treatment with first-generation SPRMs led to modification of adrenal and thyroid function and changes in prolactin secretion and because UPA binds to the glucocorticoid receptor, we report here a detailed investigation of the endocrine effects of UPA.Methods: Endocrine function was assessed...

Exogenous glucocorticoids lead to suppression of the HPA axis via negative feedback on the hypothalamus and pituitary and may be associated with increased morbidity and mortality (Wei L. Ann Intern Med. 2004). Many patients whose steroids are no longer required for their underlying disease find withdrawal challenging. Presentations: Case 1: 68-year old male treated with steroids for hepatic graft versus host disease following a stem cell transplant for EBV-lymphoproliferative ...

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...

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...