hrp0089rfc13.2 | Pituitary, Neuroendocrinology and Puberty 2 | ESPE2018

Growth Outcomes and Near Adult Height of Children with Congenital GH Deficiency (GHD) due to Abnormal Pituitary Development: Data from a Prospective, Multinational Observational Study

Child Christopher , Leger Juliane , Deal Cheri , Benabbad Imane , Jia Nan , Blum Werner

Background: Children with structural hypothalamic-pituitary anomalies, e.g. ectopic posterior pituitary (EPP) with/without pituitary stalk interruption syndrome, septo-optic dysplasia (SOD), and isolated anterior pituitary aplasia/hypoplasia (AP/HP) usually have more severe GHD and better auxological outcomes with GH therapy than those with normal hypothalamic-pituitary magnetic resonance imaging findings. However, adult height data is limited.Objective:...

hrp0082p1-d3-160 | Growth (2) | ESPE2014

Final Height and Safety Outcomes in GH-Treated Children with Short Stature Homeobox-Containing Gene (SHOX) Deficiency: Experience From a Large, Multinational, and Prospective Observational Study

Child Christopher , Quigley Charmian , Zimmermann Alan , Ross Judith , Deal Cheri , Drop Stenvert , Blum Werner

Background: To date, one randomized, controlled, clinical trial (RCCT) demonstrated that GH-treated patients with SHOX deficiency (SHOX-D) had GH-mediated height gain comparable to that of girls with Turner syndrome (TS). No new safety concerns were identified, but the study was limited by small sample size.Objective and hypotheses: To examine long-term outcomes in patients treated in standard clinical practice, we assessed final height (FH) and safety o...

hrp0084p2-412 | GH & IGF | ESPE2015

Final Height and Safety Outcomes in GH-Treated Children Born Small for Gestational Age: Experience from the Prospective GeNeSIS Observational Study

Child Chris , Quigley Charmian , Zimmermann Alan , Deal Cheri , Ross Judith , Schonau Eckhard , Blum Werner

Background: GH treatment in children born small for gestational age (SGA) has both short- and long-term growth-promoting effects and is approved in Europe and the USA (recommended dosages 0.25–0.47 mg/kg per week).Objective and hypotheses: To assess final height (FH) and safety outcomes in SGA patients receiving GH treatment in routine clinical practice using data from GeNeSIS.Method: 1208 GH-treated SGA patients were enrolled...

hrp0095fc7.2 | Growth and Syndromes | ESPE2022

Post-hoc subgroup analysis of the pivotal phase 3 study of once-weekly somatrogon vs once-daily Genotropin: results from subjects with peak stimulated growth hormone value <6.7 ng/ml

Phillip Moshe , Deal Cheri , Silverman Lawrence , Henocque Robin , Nijher Monica , Wajnrajch Michael , Wang Ronnie , Cara Jose

Objectives: Somatrogon is a long-acting recombinant human growth hormone (GH) approved by the EMA as a once weekly treatment for children with GH deficiency (GHD). The peak stimulated GH cut-off value for diagnostic criteria for GHD varies according to country-specific guidelines. The objective of this subgroup analysis of the pivotal phase 3 somatrogon study was to evaluate the primary and secondary efficacy endpoints for subjects with a peak GH value <6.7...

hrp0095t7 | Section | ESPE2022

Efficacy and safety of once-weekly somatrogon in pediatric subjects with growth hormone deficiency: lack of impact of anti-drug antibodies

Deal Cheri , Silverman Lawrence , Korth-Bradley Joan , Roland Carl , Taylor Carrie , Cara Jose , Wajnrajch Michael

Objectives: Somatrogon, a long-acting recombinant human growth hormone (GH) consisting of the amino acid sequence of human GH (hGH) and three copies of the carboxy-terminal peptide (CTP) of human chorionic gonadotropin, is approved by the EMA for treatment of children with GH deficiency (GHD). In this phase 3 study, children with GHD received either somatrogon or Genotropin. The impact of testing positive for anti-drug antibodies to somatrogon (ADA+) on the ef...

hrp0086p1-p628 | Growth P1 | ESPE2016

Height Gain and Safety Outcomes in Growth Hormone (GH)-Treated Girls and Boys with Idiopathic Short Stature (ISS): Experience from the Prospective GeNeSIS Observational Study

Child Christopher , Quigley Charmian , Zimmermann Alan , Deal Cheri , Ross Judith , Rosenfeld Ron , Cutler Jr Gordon , Blum Werner

Background: GH treatment for ISS received first approval in the USA in 2003 based on data from two controlled clinical trials. Eligibility is restricted to those with baseline (BL) height standard deviation score (HtSDS) ≤−2.25; other approvals followed, but not in Europe.Objective and hypotheses: To assess outcomes of GH therapy in a large cohort of patients (pts) treated in routine clinical practice.Methods: Short-ter...

hrp0086rfc15.6 | Late Breaking | ESPE2016

Safety of GH in Paediatrics: The GeNeSIS Prospective Observational Study Experience between 1999 and 2015 (NCT01088412)

Blum Werner , Child Christopher , Chrousos George , Cummings Elisabeth , Deal Cheri , Hasegawa Tomonobu , Holterhus Paul-Martin , Jia Nan , Lawrence Sarah , Linglart Agnes , Loche Sandro , Maghnie Mohamad , Sanchez Jacobo Perez , Polak Michel , Predieri Barbara , Richter-Unruh Annette , Rosenfeld Ron , Tajima Toshihiro , Yeste Diego , Yorifuji Tohru

Background: Although GH’s safety profile since 1987 is good, concerns remain regarding cancer (CA) risk, and French SAGhE data indicated increased mortality and cerebrovascular disease (CVD) in certain GH-treated patients (pts).Objective and hypotheses: To evaluate key safety outcome incidence in GH-treated pts of all short stature diagnoses (dx) who participated in GeNeSIS (1999–2015, 30 countries).Methods: Pt history/ca...