Endocrine Abstracts

Objectives: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by heterotopic ossification (HO) and progressive restriction of mobility. To date, no approved disease-modifying treatments for FOP exist, but interim phase III trial (NCT03312634) results suggest marked efficacy for palovarotene (PVO).1 Here, we describe methodology of the PIVOINE trial (NCT05027802) designed to allow treatment continuity and further evaluation o...

Introduction: Long-acting (LA) somatostatin analog (SSA) therapy is a common first-line medical treatment for acromegaly and NETs. There are limited real-world data on patients’ injection experience with the latest LA SSA devices/formulations.Aims: To compare the injection experience of patients with acromegaly or NETs who were receiving treatment with LAN prefilled syringe vs OCT syringe.Methods: A 2021 e-survey of adults wit...

Introduction/Background: Patients with NETs and acromegaly are commonly treated with somatostatin analogues (SSAs), such as octreotide and lanreotide depot formulations. The Pharmathen syringe is now available in several European countries and the USA for lanreotide depot injection. When using SSAs, confidence in and ease of use with syringes is important for decision-making in long-term therapy.Aims: PRESTO 3 compared nurses’ preference for the Som...

Background: CALM-NET (EudraCT 2013-002194-22; NCT02075606), a prospective, exploratory study, evaluated enumeration of pre-treatment circulating tumour cells as a predictor of symptomatic response to lanreotide autogel (LAN) in patients with functional midgut neuroendocrine tumours (NETs). Here, we describe the effect of LAN on biomarkers.Methods: Patients, ≥18 years, with functional, well- or moderately-differentiated midgut NETs were treated with...

Background: The SAGIT® instrument, designed to assist clinicians in staging and managing acromegaly, is undergoing validation. A descriptive analysis of SAGIT Validation study baseline data revealed discrepancies between investigator-evaluated disease-control status, disease activity, hormonal control, and treatment decisions in acromegaly.Objective: To describe the baseline characteristics of patients in the SAGIT® valida...

Background: In PRIMARYS, lanreotide Autogel (LAN) 120 mg/28 days reduced tumour volume (TV), and GH/IGF-1 levels in patients with GH-secreting macroadenomas. In post-hoc analyses, we investigated predictive factors for treatment responses.Methods: PRIMARYS, a 48-week, international, open-label study, involved 90 treatment-naïve patients with GH-secreting macroadenomas receiving LAN. Factors predictive for hormonal control (HC; GH ≤2.5...

Introduction: Pituitary MRI T2-signal intensity may be associated with the response to somatostatin analogue therapy in acromegalic patients. Here, we explore how best to evaluate MRI T2-signal intensity of GH-secreting pituitary macroadenoma using data from the PRIMARYS study (NCT00690898; EudraCT2007-000155-34).Methods: PRIMARYS assessed tumour volume reduction (TVR) on MRI in treatment-naïve acromegalic patients with GH-secreting pituitary macroa...

Introduction: Treatment of patients with acromegaly is complex, including surgery, pharmacotherapy and radiotherapy. The objective was to describe comorbidities and treatment patterns among patients with acromegaly in Sweden.Methods: Population-based study including all patients with a first diagnosis of acromegaly due to a pituitary adenoma in Sweden between 1 Jul 2005 and 31 Dec 2013. Data was obtained via linkage of the National Patient Register, Swed...

Background: A systematic literature review (SLR) was conducted to assess the use of independent injections (self/partner/home-administered) as an alternative to healthcare-setting injections for chronic diseases. The primary objective was to identify studies reporting on independent injection of somatostatin analogues (SSAs). Comparative evidence on independent injection of other medications was examined as a secondary objective.Methods: MEDLINE/Embase/t...

Background: Acromegaly is a rare disorder characterized by excess growth hormone (GH) and insulin-like growth factor 1 (IGF-1). Extended dosing intervals (EDIs) of pharmacological treatments may reduce patient burden and costs compared with standard dosing. This systematic literature review (SLR) investigated treatment of acromegaly at EDIs.Methods: MEDLINE/Embase/the Cochrane Library (2001–June 2021) and key congresses (2018–2021) were searche...