Endocrine Abstracts

Although diminished height velocity and short stature are important clinical markers to consider testing for GHD in children, the signs and symptoms of GHD are not always so apparent in adults. Furthermore, quality of life and metabolic health are often impacted in adults with GHD; thus, making an accurate diagnosis is important so that appropriate GH replacement therapy can be offered to these patients. However, the diagnostic work up of adult GHD can be challenging and made ...

Background: Cyclical Cushing syndrome (CCS) is a rare variant of Cushing syndrome (CS) characterized by periodic cycles of cortisol excess. Diagnosis of CCS is difficult because cortisol excess occurs unpredictably and often limited to short periods. We present 2 unusual cases where the patients developed severe prolonged life-threatening “square wave” hypercortisolemic phase followed by eucortisolemia, both phases extending > 3 months. Case 1: A 75 year old male...

Acromegaly is a rare disease most commonly caused by benign pituitary adenomas producing excess growth hormone. A profound mismatch between biological control and symptom control creates a high disease burden despite optimal disease management. To learn more about the patient experience, Acromegaly Community hosted a virtual Externally Led Patient-Focused Drug Development meeting in January 2021. There were 304 registered attendees, including 128 people with acromegaly and the...

Introduction: Subcutaneous (sc) pasireotide effectively reduces cortisol levels, improves signs/symptoms and is generally well tolerated in Cushing’s disease (CD) patients, as shown in previous clinical trials. We report interim results from a multicentre, real-world observational study evaluating the long-term safety and efficacy of pasireotide sc in clinical practice in CD patients.Methods: Adults with CD, for whom surgery has failed or is not an ...

Somapacitan is a once-weekly, long-acting growth hormone (GH) derivative approved for the treatment of adult GH deficiency (AGHD). Our objective was to evaluate the effects of somapacitan on glucose metabolism compared with daily GH or placebo in patients with AGHD using data from three previously published phase 3 trials: REAL 1 (NCT02229851), REAL 2 (NCT02382939) and REAL Japan (NCT03075644). REAL 1 was a randomised, placebo-controlled (double-blind) and active-controlled (o...

Background: Adult GHD results from insufficient growth hormone (GH) secretion from the anterior pituitary gland and may represent either a continuation of childhood-onset GHD or GHD acquired during adulthood. Clinically, adult GHD is associated with central adiposity, decreased lean muscle mass, increased fat mass, decreased bone mineral density, and reduced quality of life. Current standard of care consists of GH replacement via daily injections. Lonapegsomatropin (SKYTROFA; ...

Introduction: Growth hormone (GH) replacement therapy is safe and confers significant benefits in body composition, bone health, cardiometabolic risk factors, and quality of life in adults with GH deficiency. However, longer term safety and efficacy data are limited by the lack of consistency in data collection between studies. The study aim was to develop a simple global register by standardising and developing a minimum data set (MDS) to facilitate international collection o...