Endocrine Abstracts

Purpose: Patients with Cushing’s disease (CD) experience a wide range of physical and psychosocial health impacts. This can have a profound effect on patient’s quality of life (QOL), impairing areas such as body image, personal relationships and work performance. The CushingQoL is a valid and reliable disease-specific patient-reported outcome (PRO). When using a PRO in an interventional context it is critical to understand whether reported change is clinically meanin...

Background: In a phase 3b, open-label study (NCT02354508) assessing efficacy and safety of long-acting pasireotide in patients with uncontrolled acromegaly after ≥3 months of treatment with first-generation SSAs, 18/123 (15%) patients achieved the primary endpoint of mean growth hormone (mGH) <1.0 μg/l and insulin-like growth factor-1 (IGF-1) <ULN (upper limit of normal) at week 36. Here, we present the results from the extension phase of this study.<p c...

Introduction: Pasireotide sc has a proven favourable efficacy and safety profile in CD patients, as shown in clinical trials. Here, we report safety and efficacy results from an expanded-access study designed to allow CD patients to receive pasireotide until regulatory approval for commercial use and reimbursement was obtained in their country.Methods: Pasireotide-naïve adults with CD (mean 24-hour urinary free cortisol [mUFC; of three samples] exce...

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 ...

Acromegaly is a disorder characterized by overproduction of growth hormones (GH), which causes tissue growth in the body and comorbidities and symptoms. While prior studies examined comorbidities commonly associated with acromegaly, few have long follow-up periods necessary to characterize the long-term comorbidity profile of patients with acromegaly. There is limited literature on real-world treatment patterns of patients with acromegaly. This study describes the long-term pr...

Acromegaly is a rare disorder characterized by the overproduction of growth hormone (GH) and elevated insulin-like growth factor-1 (IGF1). While some studies have investigated the potential associations between biochemical control (i.e., normalization of IGF-1 and/or GH) and comorbidities/symptoms, few studies have long-term follow-up. This study assessed the association between biochemical control and selected comorbidities/symptoms in patients with acromegaly using real worl...

Long-term biochemical control (i.e., normalization of growth hormone [GH] and insulin-like growth factor-1 [IGF1]) is the goal of treatment of acromegaly. Few studies have characterized the sustainability of GH/IGF1 levels in acromegaly patients. This study aimed to identify long-term time trends of GH, IGF1 levels, and biochemical control in patients with acromegaly using longitudinal real world data. Medical records of adult patients with a confirmed acromegaly diagnosis, &#...