Endocrine Abstracts

Background: Pituitary neuroendocrine tumors (PitNETs) exhibiting treatment-refractory behavior are the rare subset that progress after radiation and are characterized by unrelenting growth and metastatic dissemination.Methods: To identify biomarkers of treatment-refractory behavior, two groups of patients with PitNETs were consented to matched tumor-normal whole exome and targeted next-generation DNA sequencing on a clinical protocol: a prospective group...

Background: Cushing’s syndrome (CS) has numerous comorbidities, including diabetes mellitus (DM). Levoketoconazole is a ketoconazole stereoisomer in clinical trials for treatment of CS.Methods: SONICS is a prospective, open-label, phase 3 maintenance-of-benefit study in adults with confirmed CS and mean urinary free cortisol (mUFC) of ≥1.5x upper limit of normal (ULN). Repeated hospitalization due to hyperglycemia or any complication related t...

Background: Pasireotide (Signifor®), a multireceptor-targeted somatostatin analogue, was initially approved in Europe and the USA in 2012 for treating adult patients with Cushing’s disease for whom surgery is not an option/has failed. This ‘expanded-access’ study allowed patients to receive pasireotide until regulatory approval was obtained in their country, and collected further safety/efficacy data. Here we report an interim analysis of this on...

Introduction: Long-acting pasireotide reduced urinary free cortisol (UFC) in most patients with Cushing’s disease (CD) during a large Phase III study (Lacroix et al. Lancet Diabetes Endocrinol 2018). The analyses presented here explored the impact of baseline characteristics on response to long-acting pasireotide.Methods: 150 patients with persistent, recurrent or de novo CD and mean UFC (mUFC; from three 24-hour samples collected ...

Introduction: Potent 11β-hydroxylase inhibitor osilodrostat provides cortisol level control in patients with Cushing’s syndrome (CS), as demonstrated by the LINC clinical development programme in Cushing’s disease (CD) patients.1 We report data from year 1 of the prospective observational LINC6 study (NCT05382156), evaluating long-term safety and efficacy of osilodrostat in CS patients during 3 years of routine clinical practice.<p class="abstext"...

Background: The phase 3, open-label SONICS study of levoketoconazole demonstrated sustained reduction in mean urinary free cortisol (mUFC) in adults with endogenous Cushing’s syndrome (CS). The extended evaluation phase (Ext) of SONICS reported here further assessed long-term safety, tolerability, and benefit/risk of this treatment.Methods: SONICS consisted of dose-titration (150 – 600 mg BID to attain maximally tolerated dose for mUFC normal...