hrp0095p1-513 | Growth and Syndromes | ESPE2022

Long-term effectiveness and safety of childhood growth hormone treatment in Turner syndrome from two large observational studies

Backeljauw Philippe , Pietropoli Alberto , Rohrer Tilman

Patients with Turner syndrome (TS) have short stature, despite having normal growth hormone (GH) secretion. Treatment with recombinant human GH is recommended. The effectiveness and safety of Norditropin® (somatropin, Novo Nordisk) over ≤10 years of follow-up were investigated in two non-interventional studies: NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905). Of 2,409 children with TS, 2,377 were included in the full analysis s...

hrp0094p2-280 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Outcomes in growth hormone-treated Noonan syndrome children: impact of PTPN11 mutation status

AL Jorge Alexander , Pietropoli Alberto , Kelepouris Nicky , Horikawa Reiko ,

Objectives: Mutations in PTPN11 are known to be associated with Noonan syndrome (NS), accounting for approximately 50% of cases. Data from a non-interventional and phase 3 study of Norditropin (somatropin; Novo Nordisk A/S, Denmark) were used to assess the impact of PTPN11 mutation status on growth outcomes in children with NS receiving growth hormone therapy (GHT).Methods: The ANSWER (NCT01009905) prog...

hrp0092p1-226 | Growth and Syndromes (to include Turner Syndrome) (1) | ESPE2019

Response to Growth Hormone in Very Young Children (® International Outcome Study and ANSWER Program

Rohrer Tilman R , Miller Bradley , Ostrow Vlady , Pietropoli Alberto , Polak Michel , Ross Judith

Objectives: Limited information is available on how very young children with growth hormone deficiency (GHD) respond to growth hormone (GH) replacement. We compared response to 1 year of GH therapy in children aged <2 years and prepubertal children aged ≥2 years.Methods: The two non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the ANSWER Prog...

hrp0089p2-p252 | Growth &amp; Syndromes P2 | ESPE2018

Final Results of NordiNet® International Outcome Study: Key Outcomes in Paediatric Patients

Polak Michel , Blair Jo , Rohrer Tilman R. , Pietropoli Alberto , Tonnes Pedersen Birgitte , Savendahl Lars

Background: NordiNet® International Outcome Study ([IOS]; NCT00960128), a non-interventional study (2006–2016), assessed the effectiveness and safety of real-world treatment with Norditropin®. Outcomes were assessed in children with growth hormone deficiency (GHD), born small for gestational age (SGA), Turner syndrome (TS), chronic renal disease (CRD), idiopathic short stature (ISS), Noonan syndrome (NS) and Prader-Willi syndrome (PWS)....

hrp0094p1-169 | Growth B | ESPE2021

Safety of growth hormone and cardiovascular outcomes in patients with Noonan syndrome enrolled in NordiNet® International Outcome Study (IOS) and the ANSWER Program

Romano Alicia , Kaski Juan Pablo , Dahlgren Jovanna , Kelepouris Nicky , Pietropoli Alberto , Rohrer Tilman R. , Polak Michel ,

Introduction: Growth hormone (GH) treatment has been shown to increase height velocity and adult height in patients with Noonan syndrome (NS). NS is also associated with cardiovascular (CV) anomalies, namely pulmonary stenosis and hypertrophic cardiomyopathy. Concerns persist about the role of GH in progression of CV conditions despite data, albeit limited, showing low rates of CV events and left ventricular wall thickness remaining normal. This pooled analysi...

hrp0097p1-289 | GH and IGFs | ESPE2023

Long-term effectiveness and safety of growth hormone therapy in Japanese children with short stature due to Noonan syndrome (NS): real-world data

Muroya Koji , Kawai Masanobu , Yamagishi Hiroyuki , Endo Takaaki , Pietropoli Alberto , Horikawa Reiko

Norditropin® was approved for children with short stature due to NS in Japan in 2017. The aim of this post-marketing surveillance study was to evaluate long-term safety and effectiveness of Norditropin® for the approved indication. This real-world non-interventional study (NCT03435627) was conducted February 2018–January 2022. Seventy patients enrolled: 35 received Norditropin® after study initiation (new patients); 35 were previously ra...