Searchable abstracts of presentations at key conferences in endocrinology

ea0022p556 | Neuroendocrinology and Pituitary (<emphasis role="italic">Generously supported by Novartis</emphasis>) | ECE2010

Development of a new questionnaire for evaluating QOL in adult hypopituitarism: adult hypopituitarism questionnaire (AHQ)

Shimatsu Akira , Ishii Hitoshi , Chihara Kazuo

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...

ea0081ep36 | Adrenal and Cardiovascular Endocrinology | ECE2022

Evaluation of the efficacy of osilodrostat in five patients with Cushing’s syndrome: A single-center study

Hashimoto Naoko , Kono Satomi , Kono Takashi , Akira Shimatsu , Pedroncelli Alberto , Tanaka Tomoaki

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: ...

ea0037gp.22.01 | Pituitary–Therapy of Cushing's disease | ECE2015

Phase III, multicentre, double-blind, randomised withdrawal study of osilodrostat (LCI699) in patients with Cushing's disease: a study design

Shimatsu Akira , Sauter Nicholas , Kelly Roxzana , Unge Peter , Zhi Xin , Fleseriu Maria

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) ...

ea0081rc7.5 | Rapid Communications 7: Pituitary and Neuroendocrinology 2 | ECE2022

Osilodrostat therapy improves physical manifestations of hypercortisolism in patients with cushing’s disease: findings from the phase III LINC 3 study

Pivonello Rosario , Fleseriu Maria , Akira Shimatsu , Newell-Price John , Auchus Richard , Feelders Richard , Pedroncelli Alberto , Piacentini Andrea , Biller Beverly MK

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...

ea0081p404 | Pituitary and Neuroendocrinology | ECE2022

Change in androgens and adrenal hormones during long-term osilodrostat treatment in patients with Cushing’s disease: Results from the Phase III, prospective LINC 3 study

Pivonello Rosario , M.K. Biller Beverly , Akira Shimatsu , Newell-Price John , Tabarin Antoine , Vila Greisa , Piacentini Andrea , Pedroncelli Alberto , Fleseriu Maria

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...

ea0035oc8.4 | Pituitary clinical | ECE2014

Normalization of urinary cortisol with the potent 11β-hydroxylase inhibitor LCI699 in patients with Cushing's disease: 22-week, multicentre, open-label study

Biller Beverly , Young Jacques , Hamrahian Amir , Fleseriu Maria , Molitch Mark , Pivonello Rosario , Shimatsu Akira , Shimizu Chikara , Tanaka Tomoaki , White Tracy , Hilliard Annie , Tian Chuan , Sauter Nicholas , Bertagna Xavier

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...

ea0073oc8.2 | Oral Communications 8: Pituitary and Neuroendocrinology | ECE2021

Osilodrostat is an effective and well-tolerated treatment option for patients with Cushing’s disease (CD): Final results from the LINC3 study

Fleseriu Maria , Biller Beverly , Pivonello Rosario , Akira Shimatsu , Carla Scaroni , Belaya Zhanna , Vila Greisa , Houde Ghislaine , Walia Rama , Izquierdo Miguel , Roughton Michael , Pedroncelli Alberto , Newell-Price John

IntroductionOsilodrostat, a potent oral 11β-hydroxylase inhibitor, normalized mean urinary free cortisol (mUFC) in most patients with CD during the 48-week (W) core phase of a Phase III study (LINC3: NCT02180217). We present efficacy and safety results following an extension to LINC3.MethodsCD patients with mUFC > 1.5× upper limit of normal (ULN) received osilodrostat during the core. Patients b...

ea0063oc3.1 | Cushing's and acromegaly | ECE2019

Osilodrostat provides clinical benefit over 48 weeks in patients with Cushing disease: Results from the LINC 3 study

Pivonello Rosario , Fleseriu Maria , Newell-Price John , Bertagna Xavier , Findling James , Shimatsu Akira , Gu Feng , Auchus Richard , Leelawattana Rattana , Jig Lee Eun , Hee Kim Jung , Lacroix Andre , Laplanche Audrey , O'Connell Paul , M Pedroncelli Alberto , Tauchmanova Libuse , MK Biller Beverly

Introduction: Osilodrosat is a potent oral 11β-hydroxylase inhibitor. During the 24-week, single-arm, open-label period of the Phase III LINC 3 study (NCT02180217), osilodrostat treatment demonstrated rapid, sustained reduction in mean urinary free cortisol (mUFC) in most Cushing disease (CD) patients. In the subsequent 8-week, double-blind, randomized-withdrawal phase, a significantly higher proportion of patients receiving osilodrostat maintained normal mUFC at week (W)...

ea0041ep887 | Pituitary - Clinical | ECE2016

Long-term (19-month) control of urinary free cortisol with osilodrostat in patients with Cushing’s disease: results from an extension to the LINC-2 study

Pivonello Rosario , Hatipoglu Betul , Bertagna Xavier , Fleseriu Maria , Molitch Mark E , Shimizu Chikara , Tanaka Tomoaki , Shimatsu Akira , Biller Beverly M K , Ravichandran Shoba , Kandra Albert , Sauter Nicholas , Young Jacques

Introduction: During the 22-week LINC-2 study, the potent oral 11β-hydroxylase inhibitor osilodrostat normalized UFC in 15/19 (78.9%) patients with Cushing’s disease. Most common AEs were nausea, diarrhoea, asthenia, and adrenal insufficiency. This report describes 19-month results following an extension.Methods: Patients who were receiving clinical benefit at week 22 could enter the extension. Efficacy/safety is reported for patients who enter...

ea0070oc4.5 | Pituitary and Neuroendocrinology | ECE2020

Durability of response and gender-based analysis from the LINC3 trial of osilodrostat in the treatment in cushing’s disease

Pivonello Rosario , Fleseriu Maria , Newell-Price John , Xavier Bertagna , James Findling , Akira Shimatsu , Feng Gu , Richard Auchus , Rattana Leelawattana , Jig Lee Eun , Hee Kim Jung , Andre Lacroix , Biller Beverly

Introduction: In a Phase II study, osilodrostat, a potent oral 11β-hydroxylase inhibitor, normalized mean urinary free cortisol (mUFC) in most patients with CD. We report the efficacy and safety of osilodrostat in a large CD patient population (NCT02180217).Methods: In this study, open-label osilodrostat was initiated at 2 mg bid in 137 adults with CD and mUFC > 1.5 × ULN, with dose adjustments every 2 weeks (range 1–30 mg bid) up to ...