ESPE Abstracts (2015) 84 P-2-412

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

Chris Childa, Charmian Quigleyb, Alan Zimmermannc, Cheri Deald, Judith Rosse, Eckhard Schönauf & Werner Blumg

aEli Lilly and Company, Windlesham, UK; bSydney Children’s Hospital, Randwick, NSW, Australia; cEli Lilly and Company, Indianapolis, IN, USA; dUniversity of Montreal and CHU Ste-Justine, Montreal, Canada; eDepartment of Pediatrics, Thomas Jefferson University, Philadelphia, PA, USA; fUniversity of Cologne, Children’s Hospital, Cologne, Germany; gUniversity of Giessen, University Children’s Hospital, Giessen, Germany

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 (114 Russell-Silver syndrome, 111 other known cause, 932 unknown cause). FH (defined by ≧1 of: closed epiphyses, height velocity <2 cm/year, bone age >14 years (girls)/ >16 years (boys)) was available for 203 patients (FHPop1). Subanalyses were performed for patients with baseline age ≧4/<11 years and ≧5 years GH treatment (FHPop2, N=62) and another subset with initial GH dose ≧0.2/<0.3 mg/kg pe week (FHPop3, N=26).

Results: Mean±SD baseline ages for FHPop1, FHPop2, and FHPop3 were 10.9±3.1, 8.3±1.7, and 8.9±1.9 years respectively; height SD scores (SDS) were −2.6±0.9, ±2.8±0.9, and −2.6±0.8. Initial GH dose was 0.28±0.10 mg/kg per week, increasing by ≤12% (all FHPops). FH SDS for FHPop1, FHPop2, and FHPop3 were −1.5±0.9, −1.3±0.8, and −1.1±0.8 respectively, at 16.1±1.5, 16.0±1.2, and 16.0±1.2 years of age, and after 5.0±2.9, 7.5±1.8, and 6.8±1.5 years of GH treatment; height gains were 1.1±1.0, 1.5±0.8, and 1.4±0.9 SDS respectively. ≧1 adverse event was reported for 283/1111 (25%) SGA patients vs 5 552/20 060 (28%) for all patient diagnoses in the database. Adverse events reported at ≧2.0% were precocious puberty (3%), headache, hypothyroidism, arthralgia, and ADHD (all 2%). Notable events included two deaths (displaced ventriculoperitoneal shunt; MELAS syndrome), one malignancy (lymphoma), and four diabetes cases (two type 2, one type 1, and one associated with MELAS).

Conclusion: Height gains for SGA patients in GeNeSIS were similar to those in previous studies with dosage similar to that approved in Europe; younger age at treatment start was associated with greater height gain. No new safety concerns were identified.

Conflict of interest: Author is an employee and stockholder of Eli Lilly and Company.

Funding: Sponsored by Eli Lilly and Company.

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