hrp0094p2-300 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Association of Daily Growth Hormone Injection Adherence and Height among Children with Growth Hormone Deficiency

Loftus Jane , Miller Bradley S , Parzynski Craig , Alvir Jose , Chen Yong , Jhingran Priti , Gupta Anu , DeKoven Mitch , Divino Victoria , Tse Jenny , He Jing , Wajnrajch Michael ,

Background: Use of recombinant human growth hormone (somatropin) is recommended for children with growth hormone deficiency (GHD) to normalize adult height and avoid extreme short stature. Prior research conducted among small study samples, across different study populations and health care systems, has suggested an association between adherence to somatropin and clinical outcomes, including height velocity. There is a need for further research, using real-wor...

hrp0097p1-67 | Fat, Metabolism and Obesity | ESPE2023

Percentage of Appendicular Skeletal Muscle Mass Reference and Association with Metabolic Syndrome in Korean Adolescents

Hye Lee Da , Kang Sung-Chan , Hwang Seung-sik , Jeong Lee Yun , Young Kim Hwa , Yong Lee Seong , Ho Shin Choong , Kim Jaehyun

Purpose: The association between appendicular skeletal muscle mass (ASM) and cardiometabolic risk has been emphasized. We estimated reference values of the percentage of ASM (PASM) and investigated its association with metabolic syndrome (MS) in Korean adolescents.Methods: Data from Korea National Health and Nutrition Examination Survey performed between 2009 and 2011 was used. Tables and graphs of reference PASM were ge...

hrp0095rfc7.2 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily GH in Children with GH Deficiency: The Randomized Phase 3 REAL 4 Trial

Miller Bradley , Blair Joanne , Højby Michael , Böttcher Volker , Juul Kildemoes Rasmus , Maniatis Aristides , Beck Bang Rikke , Mori Jun , Polak Michel , Stagi Stefano , Horikawa Reiko

Background: Growth hormone (GH) replacement therapy usually requires daily subcutaneous (s.c.) injections that can be burdensome for patients and their caregivers. Long-acting GH formulations aim to establish a less burdensome dosing regimen that is as safe and efficacious as daily GH to potentially improve adherence and clinical outcomes. Somapacitan, a long-acting reversible albumin-binding GH derivative, is in development for once-weekly s.c. administration...

hrp0095p2-191 | Growth and Syndromes | ESPE2022

A case report of gonadal Y-chromosome mosaicism 45, X Turner syndrome complicated by HCG-secreting gonadoblastoma

Zheng Rujiang , Ma Huamei , Liu Juncheng , Chen Huadong , Liang Jianbo , Chen Hongshan , Li Yanhong , Chen Qiuli , Zhang Jun , Guo Song , Wang Bing , Du Minlian

Objective: We report a case of a 5y3m patient who complained of breast development with "45, X Turner syndrome (TS) and HCG-secreting gonadoblastoma (Gb)" with Y chromosome mosaicism. Aim to understand the diagnosis of TS and improve the diagnosis and treatment of HCG-secreting tumors.Methods: The data of clinical diagnosis and treatment of this patient were summarized, and the literatures were reviewed.<p ...

hrp0095p2-254 | Sex Differentiation, Gonads and Gynaecology, and Sex Endocrinology | ESPE2022

Intracranial beta human chorionic gonadotropin(β-hCG)-secreting germ cell tumor in a girl with central precocious puberty

Huang Meng-tian , Zheng Ru-jiang , MA Hua-mei , LI Yan-hong , Jiang Bo , Huang Li-Bin , Guo Song , Zhang Jun , Chen Qiu-Li

Background: Precocious puberty is common in boys occasionally in girls with beta human chorionic gonadotropin (β-hCG)-secreting intracranial tumors, and is reported to be peripheral precocious puberty exclusively.Object: To described a girl with intracranial β-hCG-secreting germ cell tumor(GCT) who presented with central precocious puberty(CPP).Method:<...

hrp0086p1-p443 | Fat Metabolism and Obesity P1 | ESPE2016

The Collapse of the BDNF/POMC System in the Hypothalamus is Responsible for the Extreme Obesity with Hyperphagia Observed in Female Heterozygous MeCP2 Null Mice

Fukuhara Shota , Nakajima Hisakazu , Kodo Kazuki , Itoh Ikuyo , Shigehara Keiichi , Moroto Masaharu , Sugimoto Satoru , Mori Jun , Kosaka Kitaro , Morimoto Masafumi , Hosoi Hajime

Objective and hypotheses: The aim was to elucidate the mechanism underlying the extreme obesity observed in female heterozygous MeCP2 null mice fed a high-fat diet.Method: We examined the molecular biology and physiology of female heterozygous MeCP2 null mice (Mecp2tm1.1Bird/J, MeCP2+/− mice) fed a high-fat diet (HFD) for 12 weeks since 4 weeks of age using analytical tools. C57/BL6 mice were used as controls.<p class="abs...

hrp0082p3-d2-713 | Diabetes (1) | ESPE2014

Development of Diabetes Mellitus after Hematopoietic Stem Cell Transplantation for Childhood Leukemia

Jung In Ah , Jun Yeon Jin , Cho Won Kyoung , Lee Jae Wook , Chung Nak Gyun , Jung Min Ho , Cho Bin , Suh Byung Kyu

Background: Hematopoietic stem cell transplantation (HSCT) recipients are at risk of endocrine complications as a result of chemotherapy and radiation therapy. Development of diabetes mellitus (DM) after HSCT is less frequently reported compared with other complications.Objective and hypotheses: We investigated clinical features of newly diagnosed DM after HSCT for treatment of childhood leukemia.Method: Between April 1999 and Marc...

hrp0097fc11.1 | GH and IGFs | ESPE2023

GH replacement therapy with once-weekly somapacitan in children with GH deficiency is effective and well-tolerated: 2-year results from REAL4

Miller Bradley , Blair Joanne , Højby Rasmussen Michael , Maniatis Aristides , Mori Jun , Böttcher Volker , Bang Rikke , Polak Michel , Horikawa Reiko

Daily subcutaneous (s.c.) injections of growth hormone (GH) to treat GH deficiency (GHD) in children is burdensome for both patients and caregivers. Somapacitan (Novo Nordisk) is a long-acting reversible albumin-binding human GH derivative in development for once-weekly s.c. administration in children with GHD, and aims to overcome the treatment burden of daily injections. REAL4 is a multi-national, randomised, open labelled phase 3 trial with a 52-week main phase followed by ...

hrp0097fc14.5 | Late Breaking | ESPE2023

Predicting Average IGF-I Concentration for Once-Weekly Somapacitan in Children with Growth Hormone Deficiency

F. Backeljauw Philippe , Kamal Lyauk Yassine , C. Blair Joanne , S. Miller Bradley , Mori Jun , Højby Rasmussen Michael , Juul Kildemoes Rasmus

Growth hormone (GH) stimulates insulin like growth factor I (IGF I) release. IGF-I is the standard biomarker for monitoring GH effects during treatment and to achieve optimal long-term safety, and to a limited extent, monitor efficacy in children with GH deficiency (GHD). The IGF I profile during treatment with a long acting GH (LAGH), such as once weekly somapacitan (Novo Nordisk), differs from the daily GH profile by exhibiting larger peaks and troughs over the dosing interv...

hrp0097p1-100 | GH and IGFs | ESPE2023

Exploring healthcare professionals’ attitudes towards digitalization and the perceived usefulness and ease of use of digital solutions in patients receiving growth hormone therapy: Results of a Korean participatory study

Wook Chae Hyun , Rivera Romero Octavio , Kun Cheon Chong , Sang Lee Hae , Kim Jihyun , Eun Moon Jung , Koledova Ekaterina , Sil Oh Eun , Yang Yoo-Jin , Rhie Young-Jun

Background: Aluetta® Smartdot™ (Merck Healthcare KGaA, Darmstadt, Germany) is a novel injection device for administering recombinant-human growth hormone (r-hGH), integrated with a smart knob attachment for data transmission that combines ease of use with advanced capabilities. Integration with Growzen™ digital ecosystem empowers healthcare professionals (HCPs) with remote monitoring of patient adherence, thereby promoting optimal cl...