Abstract Search

hrp0098p1-280 | Sex Endocrinology and Gonads 3 | ESPE2024

Validation Of A New Short Parent Reported Outcomes (PRO) Questionnaire For Boys With A Condition Affecting Sex Development (CSD)

Tseretopoulou Xanthippi , R. Ali Salma , Gardner Melissa , Flett Martyn , Lee Boma , O'Toole Stuart , Steven Mairi , E. Sandberg David , Ahmed S.Faisal

Background: To aid assessment of parent-reported quality of life outcomes (PRO) in routine clinical setting in young children with a condition affecting sex development, a short questionnaire (PRO-CSD) that includes a parent-proxy report (PPR) and a parent-self-report measure (PSR) has been recently developed and requires further validation. Methods: Parents of 98 boys with a median age of 2.9 yrs (range, 0.2,6.5) and me...

0 shares

hrp0095p1-582 | Sex Differentiation, Gonads and Gynaecology, and Sex Endocrinology | ESPE2022

An International Study Of The Association Between Local Health Care Resources And Acute Adrenal Insufficiency Events In Children With Congenital Adrenal Hyperplasia

Tseretopoulou Xanthippi , R. Ali Salma , Bryce Jillian , Navoda Atapattu , H. Birkebaek Niels , Baronio Federico , Bonfig Walter , L. Claahsen-van der Grinten Hedi , Cools Martine , Darendeliler Feyza , Poyrazoglu Sukran , de Sanctis Luisa , Elsedfy Heba , J.J. Finken Martijn , E. Fluck Christa , Gevers Evelien , Korbonits Marta , Guran Tulay , Guven Ayla , A. Hughes Ieuan , Tadokoro-Cuccaro Rieko , Thankamony Ajay , Iotova Violeta , Krone Ruth , Lichiardopol Corina , B. Mendonca Berenice , A. S. S. Bachega Tania , C. Miranda Mirela , Milenkovic Tatjana , Mohnike Klaus , Nordenstrom Anna , J. van der Kamp Hetty , Faisal Ahmed Syed

Introduction: The reported occurrence and management of acute adrenal insufficiency (AI) – related adverse events (AE) vary widely between centres and may depend on available resources.Aim: To use the data within the I-CAH Registry to understand the association between the occurrence and management of AI related AE, their management and the availability of local resources.Methods:</st...

0 shares

hrp0097fc1.5 | Adrenals and HPA Axis | ESPE2023

Management of congenital adrenal hyperplasia in the first 90 days of life: a multi-centre I-CAH analysis of contemporary practice

BN Lim David , Bryce Jillian , R Ali Salma , Tseretopoulou Xanthippi , H Birkebaek Niels , E Hannema Sabine , Campos-Martorell Ariadna , Clemente Maria , Neumann Uta , E Flück Christa , Metzger Sara , E Krone Ruth , German Alina , Baronio Federico , Atapattu Navoda , N Seneviratne Sumudu , Cools Martine , Mieke Théry , Guran Tulay , Yavas Abali Zehra , Fu Antony , Janus Dominika , Wasniewska Malgorzata , Coco Roberto , Russo Gianni , Rita Stancampiano Marianna , Bonfig Walter , Salerno Mariacarolina , L Claahsen-Van Der Grinten Hedi , PH Adriaansen Bas , Bachega Tania , Cavalieri Costa Fernanda , Marginean Otilia , De Sanctis Luisa , Probst Ursina , Lenherr-Taube Nina , Konrad Daniel , O’Connell Michele , Gawlik Aneta , E Sandberg David , Shnorhavorian Margarett , Krone Nils , Faisal Ahmed S , H Davies Justin

Aims: Historical I-CAH data shows considerable variation in the management of 21-hydroxylase deficiency (21-OHD) congenital adrenal hyperplasia (CAH) in infancy despite existence of several guidelines. Using the I-CAH registry we analysed contemporary early infancy natural history data, creating benchmarks as part of continuous quality improvement.Methods: Of 136 infants born in 2018-2023 and treated for 21-OHD CAH withi...

2 shares

hrp0097p1-3 | Adrenals and HPA Axis | ESPE2023

Acute Adrenal Insufficiency Related Adverse Events In Children With Congenital Adrenal Hyperplasia (CAH): Changes During The Period 2019-2022 In I-CAH

Tseretopoulou Xanthippi , R Ali Salma , Bryce Jillian , Amin Nadia , Atapattu Navoda , Bachega Tania , Baronio Federico , H Birkebaek Niels , Bonfig Walter , H Davies Justin , Thomas Tessy , de Vries Liat , Elsedfy Heba , Hussein Amr Nermine , E. Flueck Christa , Globa Evgenia , Zelinska Natalia , Guran Tulay , Yavas Abali Zehra , Guven Ayla , E. Hannema Sabine , Iotova Violeta , Konrad Daniel , P. Krone Nils , Lenherr-Taube Nina , Leka-Emiris Sofia , Vlachopapadopoulou Elpis , R. Lichiardopol Corina , Marginean Otilia , L. Markosyan Renata , Niedziela Marek , Banaszak-Ziemska Magdalena , Neumann Uta , Phan-Hug Franziska , Polyrazoglu Sukran , Probst Ursina , Randell Tabitha , Russo Gianni , Salerno Mariacarolina , Shnorhavorian Margarett , van Eck Judith , Vieites Ana , Wasniewska Malgorzata , F Ahmed Syed

Background: In 2019, the International CAH Registry (I-CAH) performed a benchmarking exercise of acute adrenal insufficiency related adverse events including adrenal crises (AC) and sick day episodes (SDE).Methods: In 2022, I-CAH data on children aged <18 years at first visit with 21-hydroxylase deficiency CAH from 35 centres in 19 countries were analysed to examine the current occurrence of SDE and AC and compare it ...

0 shares

hrp0095p1-3 | Adrenals and HPA Axis | ESPE2022

Blood pressure (BP) status in Congenital Adrenal Hyperplasia (CAH) – longitudinal analysis of real world data from the I-CAH registry

R. Lawrence Neil , Bacila Irina , Dawson Jeremy , R. Ali Salma , LT van den Akker Erica , Aparecida Sartori Sanchez Bachega Tânia , Baronio Federico , Holtum Birkebæk Niels , Bonfig Walter , Claahsen van der Grinten Hedi , Cools Martine , Correa Costa Eduardo , 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 , Raducanu Lichiardopol Corina , L. Markosyan Renata , Milenkovic Tatjana , Costa de Miranda Mirela , Neumann Uta , Newell-Price John , Poyrazoğlu Şükran , Probst-Scheidegger Ursina , Russo Gianni , De Sanctis Luisa , Safwaan Adam , Seneviratne Sumudu , Rita Stancampiano Marianna , Tadokoro-Cuccaro Rieko , Thankamony Ajay , Vieites Ana , Wasniewska Malgorzata , Yeste Diego , Daniel Eleni , Tomlinson Jeremy , Faisal Ahmed S. , Krone Nils

Introduction: Data on BP in children with classic congenital adrenal hyperplasia (CAH) is inconsistent.Aim: To assess the trend in BP throughout childhood using data from the International CAH registry and compare this to normative data from the American Academy of Paediatrics (AAP).Method: This retrospective multi-centre study included 37 centres from 20 countries. BP data was ana...

4 shares

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...

0 shares

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...

0 shares

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...

0 shares

ESPE Abstracts

Validation Of A New Short Parent Reported Outcomes (PRO) Questionnaire For Boys With A Condition Affecting Sex Development (CSD)

An International Study Of The Association Between Local Health Care Resources And Acute Adrenal Insufficiency Events In Children With Congenital Adrenal Hyperplasia

Management of congenital adrenal hyperplasia in the first 90 days of life: a multi-centre I-CAH analysis of contemporary practice

Acute Adrenal Insufficiency Related Adverse Events In Children With Congenital Adrenal Hyperplasia (CAH): Changes During The Period 2019-2022 In I-CAH

Blood pressure (BP) status in Congenital Adrenal Hyperplasia (CAH) – longitudinal analysis of real world data from the I-CAH 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).

BiosciAbstracts