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hrp0098p1-271 | Pituitary, Neuroendocrinology and Puberty 4 | ESPE2024

The Use Of GloBE-Reg As A Global Platform For Performing Safety & Efficacy Studies Of rhGH Therapy

Alimussina Malika , Bryce Jillian , Chen Minglu , Koley Sanhita , E Al-Agha Abdulmoein , Amin Nadia , Atapattu Navoda , C Chan Kate , Chen Jiajia , Ki Chung Ying , Deyanova Yana , Fu Antony , Gong Chunxiu , Iotova Violeta , Janus Dominika , AL Jorge Alexander , Markosyan Renata , Maniatis Aristides , Seneviratne Sumudu , M Shaikh Guftar , Shepherd Sheila , JW Tack Lloyd , Wasniewska Malgorzata , WC Wong Sammy , Yu Wai Wai , Ching Chen Suet , Faisal Ahmed S

Introduction: The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions (GloBE-Reg, https://globe-reg.net/) project was launched in 2022 with the aim of supporting studies that focus on effectiveness and long-term safety of specific therapies. The project’s initial focus has been on recombinant human growth hormone therapy (rhGH) given that there are existing gaps in knowledge with the introdu...

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hrp0098rfc5.6 | Growth and Syndromes | ESPE2024

Pubertal induction in girls with Turner syndrome – retrospective data from the International TS registry

Gawlik-Starzyk Aneta , Matthews Debbie , Błaszczyk Ewa , Brain Caroline , Öhman Kriström Berit , M McGlacken-Byrne Sinéad , Sas Theo , van der Velden Janielle , Verlinde Franciska , Wasniewska Malgorzata , Smith Arlen , Gawlik Jakub , Atapattu Navoda , Bertelloni Silvano , Binder Gerhard , Cheetham Tim , L Claahsen-van der Grinten Heidi , M O'Connell Susan , Cools Martine , Dirlewanger Mirjam , Elsedfy Heba , A Baky Fahmy Mohamed , Fica Simona , E. Flück Christa , F Gevers Evelien , Globa Evgenia , Guazzarott Laura , Guven Ayla , E. Hannema Sabine , Herrmann Gloria , Howard Sasha , Iotova Violeta , Januś Dominika , Konrad Daniel , Krone Nils , Kyrilli Aglaia , Leka-Emiri Sofia , Marginean Otilia , Markosyan Renata , Niedziela Marek , Nordenstrom Anna , Obara - Moszynska Monika , Poyrazoglu Sukran , Probst Ursina , Rohayem Julia , Russo Gianni , Šandrk Beslać Marija , Schwitzgebel Valerie , Nimali Seneviratne Sumudu , Shenoy Savitha , Starzyk Jerzy , Tourlamain Gilles , Lenherr Taube Nina , Ucar Ahmet , Utari Agustini , Wędrychowicz Anna , Wojtyś Joanna , Zainaba Mohamed , Bryce Jillian , Chen Minglu , Koley Sanhita , Alimussina Malika , N.S. Matthews John , Faisal Ahmed Syed , D.C. Donaldson Malcolm

Background: Current guidelines for girls with gonadal dysgenesis due to Turner Syndrome (TS) recommend initiating estrogen therapy at 11-12 years of age, using 'natural' 17-β estradiol. However, there is scant evidence regarding the optimal modality of induction, particularly concerning whether oral or transdermal routes are more effective or acceptable.Aim: To retrospectively evaluate differences in puber...

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hrp0098p1-6 | Adrenals and HPA Axis 1 | ESPE2024

Impact of Fludrocortisone on Blood Pressure in Young Children Under 5 with CAH in the I-CAH Registry: Autoregressive Causal Inference Analysis

R. Lawrence Neil , Bacila Irina , Tonge Joseph , Dawson Jeremy , S. Collins Gary , Lang Zi-Qiang , Bryce Jillian , Alimussina Malika , Chen Minglu , R. Ali Salma , Adam Safwaan , L.T. van den Akker Erica , A.S.S. Bachega Tânia , Baronio Federico , H. Birkebæk Niels , Bonfig Walter , Claahsen–van der Grinten Hedi , Cools Martine , C. Costa Eduardo , Debono Miguel , de Vries Liat , Flück Christa , Gazdagh Gabriella , Güven Ayla , Hannema Sabine , Iotova Violeta , J. van der Kamp Hetty , Krone Ruth , Leka-Emiri Sofia , Clemente-León María , R. Lichiardopol Corina , L. Markosyan Renata , Milenkovic Tatjana , C. Miranda Mirela , Neumann Uta , Newell-Price John , Poyrazoğlu Şükran , Probst-Scheidegger Ursina , Rey Rodolfo , Russo Gianni , De Sanctis Luisa , N. Seneviratne Sumudu , R. Stancampiano Marianna , Tadokoro-Cuccaro Rieko , Thankamony Ajay , Vieites Ana , Wasniewska Malgorzata , Yeste Diego , Tomlinson Jeremy , Faisal Ahmed S , Krone Nils

Background: The International Congenital Adrenal Hyperplasia (CAH) Registry provides rich longitudinal data from CAH patients with 21 Hydroxylase deficiency. We estimated the average total causal effect of fludrocortisone on blood pressure in patients with CAH by modelling within a causal inference framework.Methods: We constructed a directed acyclic graph (DAG) with domain experts (using Dagitty.net) to establi...

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hrp0098p1-7 | Adrenals and HPA Axis 1 | ESPE2024

Variation in Blood Pressure in Young Adults with 21a-Hydroxylase Deficiency: Longitudinal Multilevel Modelling of Data from the I-CAH Registry

R. Lawrence Neil , Bacila Irina , Tonge Joseph , Dawson Jeremy , S. Collins Gary , Lang Zi-Qiang , Bryce Jillian , Alimussina Malika , Chen Minglu , R. Ali Salma , Adam Safwaan , L.T. van den Akker Erica , A.S.S. Bachega Tânia , Baronio Federico , Bonfig Walter , Claahsen-van der Grinten Hedi , Cools Martine , C. Costa Eduardo , Debono Miguel , de Vries Liat , Flück Christa , Gazdagh Gabriella , Güven Ayla , Hannema Sabine , Iotova Violeta , J van der Kamp Hetty , Krone Ruth , Leka-Emiri Sofia , Clemente-León María , R. Lichiardopol Corina , L. Markosyan Renata , Milenkovic Tatjana , C. Miranda Mirela , Neumann Uta , Newell-Price John , Poyrazoğlu Şükran , Probst-Scheidegger Ursina , Russo Gianni , De Sanctis Luisa , N. Seneviratne Sumudu , R. Stancampiano Marianna , Tadokoro-Cuccaro Rieko , Thankamony Ajay , Vieites Ana , Wasniewska Malgorzata , Yeste Diego , Tomlinson Jeremy , Faisal Ahmed S , Krone Nils

Background: Alterations in blood pressure in treated CAH due to 21 hydroxylase deficiency is contentious, with studies reporting different effects. We used data from young adults entered into the I-CAH registry to assess the change in blood pressure readings recorded within patients over time.Methods: We used longitudinal mixed effects modelling (LMEM) in R to account for repeated measures in two levels within patients a...

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hrp0098fc4.1 | Adrenals and HPA Axis 1 | ESPE2024

Real world data analysis of contemporary therapy and its association to anthropometric outcomes in 1500 patients with congenital adrenal hyperplasia (CAH).

A Bacila Irina , R Lawrence Neil , Balagamage Chamila , Bryce Jillian , R Ali Salma , Alimussina Malika , Chen Minglu , Bhushan Arya Ved , Atapattu Navoda , A Bachega Tânia , Barat Pascal , Baronio Federico , Bertelloni Silvano , Birkebæk Niels , Bonfig Walter , de Bruin Christiaan , Claahsen – van der Grinten Hedi , Cools Martine , Costa Eduardo , Crowne Elizabeth , H Davies Justin , Debono Miguel , de Vries Liat , Dumic Kubat Katja , Elsedfy Heba , Evliyaoglu Olcay , Flüeck Christa , Gazdagh Gabriella , German Alina , F Gevers Evelien , Globa Evgenia , Guran Tulay , Güven Ayla , Guazzarotti Laura , Hannema Sabine , Hatipoglu Nihal , Janus Dominika , van der Kamp Hetty , Karabouta Zacharoula , Krone Ruth , Lenherr Taube Nina , Leka-Emiri Sofia , Lichiardopol Corina , Mamdouh Hassan Mona , Marginean Otilia , Markosyan Renata , Mazen Inas , Miles Harriet , L Mitchell Anna , Mohnike Klaus , Neumann Uta , Niedziela Marek , Nordenstrom Anna , M O’Connell Susan , Phan-Hug Franziska , Poyrazoglu Sukran , Probst-Scheidegger Ursina , Rey Rodolfo , Russo Gianni , Salerno Mariacarolina , de Sanctis Luisa , N Seneviratne Sumudu , Segev-Becker Anat , Shenoy Savitha , Skae Mars , Thankamony Ajay , Ucar Ahmet , Utari Agustini , Vieites Ana , Wasniewska Malgorzata , Faisal Ahmed S , P Krone Nils

Introduction and Objective: Previous evidence from the I-CAH registry showed wide variation of glucocorticoid (GC) replacement between different countries and centres. We wanted to explore the impact of different GC doses on height and weight in children and young people with CAH.Methods: We analysed data from patients under 18 years with 21-hydroxylase deficiency recorded in the I-CAH registry from assessments since 200...

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ESPE Abstracts

The Use Of GloBE-Reg As A Global Platform For Performing Safety & Efficacy Studies Of rhGH Therapy

Pubertal induction in girls with Turner syndrome – retrospective data from the International TS registry

Impact of Fludrocortisone on Blood Pressure in Young Children Under 5 with CAH in the I-CAH Registry: Autoregressive Causal Inference Analysis

Variation in Blood Pressure in Young Adults with 21a-Hydroxylase Deficiency: Longitudinal Multilevel Modelling of Data from the I-CAH Registry

Real world data analysis of contemporary therapy and its association to anthropometric outcomes in 1500 patients with congenital adrenal hyperplasia (CAH).

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