Endocrine Abstracts

Objective: To develop and validate the adult hypopituitarism questionnaire (AHQ) as a disease-specific, self-administered questionnaire for evaluation of QOL in adult patients with hypopituitarism.Methods: The development and validation of a new questionnaire were performed in a standardized manner: item development, pilot-testing and psychometric validation.Result: Content validity of the initial questionnaire was evaluated via tw...

Background and objective: The number of reports of metastatic (secondary) pituitary tumors has been increasing with progression of diagnostic and treatment skills for cancer patients. However, there were no nation-wide epidemiologic surveys or epidemiologic reviews about this topic. We have started a multi-institutional joint research in Japan to understand clinical features of metastatic pituitary tumors.Subjects and methods: We distributed 1069 closely...

Context: Osilodrostat (Osi), a potent inhibitor of 11β-hydroxylase, blocks the conversion of 11-deoxycortisol to cortisol and improves hypercortisolism in patients with Cushing’s syndrome (CS). Here, we report a study evaluating the efficacy of Osi in five non-Cushing’s disease (CD) CS patients treated with Osi in Japan.Subjects and Results: Five patients with non-CD CS were treated with Osi at Chiba University [primary disease breakdown: ...

Introduction: Osilodrostat is a potent, oral inhibitor of 11β-hydroxylase, the enzyme that catalyses final step of cortisol biosynthesis. In a phase II study, 15/19 patients treated with osilodrostat met the primary endpoint (normal urinary free cortisol (UFC) at 22 weeks); osilodrostat was generally well tolerated. This phase III study aims to confirm the efficacy and long-term safety of osilodrostat.Patients and methods: Adults (18–75 years) ...

Primary aldosteronism (PA) is the most common cause of secondary hypertension. Although decline in renal function especially that experienced after adrenalectomy (ADX) has been demonstrated, details of the mechanism remain to be elucidated. Aim of the study was to investigate the factors predicting renal outcome after ADX in PA. Twenty patients with PA and four patients with non-functioning adrenal tumor (NFT) as control were studied. eGFR, serum potassium, plasma aldosterone ...

Objective: Pseudohypoparathyroidism type 1b (PHP-1b) is rare disorders resulting from genetic and epigenetic aberrations in the GNAS locus. PHP-1b usually defined by isolated renal resistance to PTH, is due to a maternal loss of GNAS exonA/B methylation, leading to decreased Gs’α expression in specific tissues. To clarify the usefulness of methylation specific multiplex ligation-dependent probe amplification (MS-MLPA), we evaluate the genetic and epigenetic changes o...

Inflammatory lesions of the pituitary gland are rarely encountered. Recently, the concept of IgG4-related systemic disease was proposed and several cases associated with infundibulo-hypophysitis were reported. We report 4 additional cases and review the cases in the literature. Case 1: 75-year-old female had diabetes insipidus due to infundibulo-neurohypophysitis for 25 years and chronic thyroiditis. She developed obstructive jaundice and surgery showed autoimmune pancreatitis...

Background: Improving physical manifestations of hypercortisolism is an important treatment goal for patients with Cushing’s disease (CD). In the Phase III LINC 3 study (NCT02180217), osilodrostat therapy, a potent oral 11β-hydroxylase inhibitor, rapidly normalised mean urinary free cortisol (mUFC) in most patients with CD and sustained control of mUFC over a median treatment period of 130 weeks (W). Here we describe concomitant improvements in physical manifestation...

Introduction: Osilodrostat decreases cortisol production by inhibiting 11β-hydroxylase, increasing adrenal hormones above the blockade. Here, we describe these effects of osilodrostat and associated adverse events (AEs). The efficacy and safety of osilodrostat in patients with Cushing’s disease (CD) were confirmed in the published Phase III, prospective LINC 3 study (NCT02180217).Methods: 137 patients with CD (mUFC >1.5x upper limit of norm...

Background: A proof-of-concept study (LINC 1) demonstrated that after 10 weeks, LCI699 normalized UFC in 11/12 patients with Cushing’s disease. This interim analysis of the first eight patients enrolled into a longer-term study (LINC 2) further evaluates LCI699 in Cushing’s disease; the full analysis on all 19 enrolled patients is expected in time for the congress.Methods: There were two study groups. Previous LINC 1 participants (follow-up coh...