hrp0095rfc10.5 | GH and IGFs | ESPE2022

Evaluation of health-related quality of life & problem behavior in adults born small for gestational age treated with growth hormone during childhood in comparison with untreated controls

Dorrepaal Demi , Wesley Goedegebuure , der Steen Manouk van , Anita Hokken-Koelega

Background: Being born small for gestational age (SGA) has been associated with a reduction in health-related quality of life (HRQoL) and more problem behaviour. Growth hormone (GH) treatment, which leads to a significant improvement in adult height (AH), improves HRQoL and decreases problem behavior in SGA children. However, such results are very limited in SGA adults who were treated with GH during childhood.Objective:...

hrp0097fc11.4 | GH and IGFs | ESPE2023

Long-term GH-treatment of children born small for gestational age (SGA) does not result in cerebrovascular abnormalities in adulthood compared to untreated controls

Dorrepaal Demi , Goedegebuure Wesley , Smagge Lucas , van der Steen Manouk , van der Lugt Aad , Hokken-Koelega Anita

Background: Increased cerebrovascular morbidity and mortality was reported in adults who were treated with growth hormone (GH) during childhood, including those born SGA, compared to the general population. However, previous studies did not have an appropriate control group of untreated SGA adults which was a major limitation.Objective: To assess cerebrovascular abnormalities (aneurysms, previous intracerebral hemorrhage...

hrp0097fc11.2 | GH and IGFs | ESPE2023

Clinical characteristics of heterozygous ACAN gene variants and longer-term response to growth hormone treatment: real-world data

Renes Judith , Reedijk Ardine , Losekoot Monique , Kant Sarina , van der Steen Manouk , van der Kaay Danielle , Hokken-Koelega Anita , van Duyvenvoorde Hermine , de Bruin Christiaan

Background: Heterozygous pathogenic variants in the ACAN gene underlie disproportionate short stature with characteristically accelerated bone age (BA) maturation and/or osteochondritis dissecans (OD)/early-onset osteoarthritis (OA).Objective: To describe the phenotypic spectrum and assess the response and safety of growth hormone (GH) treatment in children with a heterozygous pathogenic ACAN variant.</...