hrp0097lb17 | Late Breaking | ESPE2023

The Majority of Children with Pediatric Growth Hormone Deficiency Treated With Lonapegsomatropin for Up To 6 Years Met or Exceeded Average Parental Height SDS: Final Results of enliGHten

Vlachopapadopoulou Elpis , Thornton Paul , Hofman Paul , Nadgir Ulhas , Saenger Paul , Malievskiy Oleg , Aghajanova Elena , Korpal-Szczyrska Maria , Mao Meng , Abdelrahman Sohair , Komirenko Allison , Shu Aimee , Maniatis Aristides

Introduction: Lonapegsomatropin (SKYTROFA, TransCon hGH), a prodrug of somatropin administered once-weekly, is approved for the treatment of pediatric growth hormone deficiency (GHD) by the Food and Drug Administration and European Commission. In the pivotal 52-week phase 3 heiGHt trial and 26-week fliGHt trial (enrolled treatment-naïve and treatment-experienced participants, respectively), lonapegsomatropin demonstrated safety and efficacy in children wi...

hrp0092fc14.4 | GH and IGF4 | ESPE2019

Once-Weekly TransCon hGH vs. Daily hGH in Pediatric Growth Hormone Deficiency: The Phase 3 heiGHt Trial

Vlachopapadopoulou Elpis , Aghajanova Elena , Chertok Elena , Korpal-Szczyrska Maria , Giorgadze Elene , Kovalenko Tatiana , Maniatis Aristedes , Thornton Paul , Hofman Paul , Song Wenjie , Shu Aimee , Karpf David , Beckert Michael , Leff Jonathan

Background: TransCon hGH is a sustained-release prodrug in development as a long-acting GH for children with growth hormone deficiency (GHD). TransCon hGH consists of a parent drug, growth hormone (hGH; somatropin), that is transiently bound to a carrier via a TransCon linker. The carrier extends hGH circulation time in the body and fully active hGH is released over one week at physiologic pH and temperature. Unlike other molecules in development, TransCon hGH...

hrp0092fc7.4 | Diabetes and Insulin Session 2 | ESPE2019

Metabolic Syndrome Features in Pre-Pubertal Children Born After Maternal Pre-Eclampsia

Goffin Sarah , Derraik José , Chiavaroli Valentina , Biggs Janene , Hofman Paul , Groom Katie , Cutfield Wayne

Background: Pre-eclampsia is associated with important complications for both mother and baby in the short term, but there are limited data about its long-term effects on offspring metabolism. Thus, we aimed to assess whether maternal pre-eclampsia was associated with adverse effects on metabolism and body composition in the offspring in childhood.Methods: We studied healthy pre-pubertal children (aged 4–10 years) b...

hrp0086rfc4.4 | Pathophysiology of Obesity | ESPE2016

Metabolic Syndrome Markers Correlate with Gut Microbiome Activity in Children Born Very Preterm

Chiavaroli Valentina , Jayasinghe Thilini N , Ekblad Cameron , Derraik Jose , Hofman Paul , O'Sullivan Justin , Cutfield Wayne

Background: Fifteen years ago children born very preterm (<32 weeks of gestation) were reported to be insulin resistant. Neonatal intensive care has since improved considerably, but it is unclear whether this has affected long-term outcomes in those born preterm. Abnormalities in gut microbiome, which influence host metabolism, have been found in preterm newborns.Objective and hypotheses: We aimed to assess whether children born very preterm still ha...

hrp0086p1-p455 | Fat Metabolism and Obesity P1 | ESPE2016

Seven-year Follow-up of Mothers from a Randomized Controlled Trial of Exercise in Pregnancy and their Offspring

Chiavaroli Valentina , Hopkins Sarah , Derraik Jose , Seneviratne Sumudu , Biggs Janene , Rodrigues Raquel , Cutfield Wayne , Hofman Paul

Background: We have previously shown in a randomised controlled trial that moderate-intensity exercise over the last 20 weeks of gestation in healthy nulliparous women led to a birth weight reduction of approximately 250 g.Objective and hypotheses: We aimed to assess the long-term effects of exercise in pregnancy on anthropometry and body composition in mothers and offspring 7 years after the intervention. We hypothesized that women who exercised in preg...

hrp0084p2-215 | Bone | ESPE2015

Whole-Body Vibration Training Improves Physical Function and Increases Bone and Muscle Mass in Youngsters with Mild Cerebral Palsy

Gusso Silmara , Colle Patricia , Derraik Jose G B , Biggs Janene , Munns Craig , Cutfield Wayne , Hofman Paul

Background: Adolescents with cerebral palsy (CP) have decreased muscle mass resulting in impaired mobility and osteopenia. There is a void in therapeutic interventions aimed at increasing muscle mass, muscle function as well as osteopoenia in this population. Whole body vibration training (WBVT) has the potential to fill this therapeutic void by maintaining/increasing muscle mass and bone mineral accrual during growth.Objective and hypotheses: We aimed t...

hrp0084p2-408 | GH &amp; IGF | ESPE2015

Does Skeletal Disproportion in Children with Idiopathic Short Stature Influence Response to GH Therapy?

Cutfield Wayne S , Lindberg Anders , Hofman Paul , Derraik Jose , Geffner Mitchell E , Camacho-Hubner Cecilia

Background: Children with ISS have an array of causes that lead to short stature and/or poor growth velocity. Genetic causes of short stature, notably SHOX mutations, can be associated with subtle skeletal disproportion with shorter limbs, manifesting as increased sitting-to-standing height ratios or SDS.Objective and hypothesis: Children with ISS and skeletal disproportions have a diminished growth response to GH treatment compared to chi...

hrp0092p1-50 | Fat, Metabolism and Obesity | ESPE2019

Two-year Outcomes of Whanau Pakari: A Novel Home-based Intervention for Child and Adolescent Obesity

Anderson Yvonne , Wynter Lisa , O'Sullivan Niamh , Wild Cervantée , Grant Cameron , Cave Tami , Derraik José , Hofman Paul

Background: Whilst multi-disciplinary intervention models for children and adolescents with obesity remain recommended best practice, there is a lack of long-term outcome data, especially in home-based models and programs embedded within the clinical setting. Whānau Pakari is a community-based multi-disciplinary assessment/intervention programme for child obesity, focused on reducing health inequity. Based in Taranaki, Aotearoa/New Zealand, it focusses on...

hrp0089p1-p115 | Fat, Metabolism and Obesity P1 | ESPE2018

Greater Maternal BMI Early in Pregnancy and Excessive Gestational Weight Gain are Independently Associated with Adverse Health Outcomes in the Offspring at Age 7 Years

Derraik Jose G B , Chiavaroli Valentina , A Hopkins Sarah , Biggs Janene B , Rodrigues Raquel O , Seneviratne Sumudu N , McCowan Lesley M E , Cutfield Wayne S , Hofman Paul L

Background: Maternal overweight/obesity during pregnancy and excessive gestational weight gain (GWtG) have been recognized as important early-life risk factors for childhood obesity. We aimed to examine whether maternal BMI at 20 weeks of gestation and excessive GWtG were associated with alterations in body composition and metabolism in childhood in the offspring of primiparous mothers who participated in a randomised controlled trial of exercise regimen during pregnancy.<...

hrp0094p1-161 | Growth B | ESPE2021

Continued Safety and Efficacy of Weekly Lonapegsomatropin (TransCon hGH) for up to Two Years in Children with Growth Hormone Deficiency (GHD)

Aghajanova Elena M. , Casella Samuel J. , Nadgir Ulhas , Hofman Paul , Saenger Paul , Song Wenjie , Mao Meng , Chessler Steven , Komirenko Allison S. , Beckert Michael , Shu Aimee D. , Thornton Paul S. , Maniatis Aristides K. ,

Lonapegsomatropin (TransCon hGH) is an investigational once-weekly prodrug of somatropin for the treatment of GHD. Previous trials in treatment-naïve (52-week heiGHt Trial) and treatment-experienced children (26-week fliGHt Trial) have reported the efficacy and safety of lonapegsomatropin. Subjects were eligible to enter the open-label extension enliGHten Trial, which continues to evaluate weekly lonapegsomatropin in pediatric GHD. In heiGHt, treatment-naïve subjects...