Efficacy and Safety Results of Long-Term Growth Hormone Treatment of Idiopathic Short Stature

• Published in: The journal of clinical endocrinology and metabolism 2005-09, Vol.90 (9), p.5247-5253
• Main Authors: Kemp, Stephen F., Kuntze, Joyce, Attie, Kenneth M., Maneatis, Thomas, Butler, S., Frane, James, Lippe, Barbara
• Format: Article
• Language: English
• Subjects: Adolescent; Biological and medical sciences; Body Height - drug effects; Child; Cohort Studies; Drug Administration Schedule; Endocrinopathies; Female; Fundamental and applied biological sciences. Psychology; Growth Disorders - drug therapy; Human Growth Hormone - administration & dosage; Human Growth Hormone - adverse effects; Human Growth Hormone - therapeutic use; Humans; Male; Medical sciences; Product Surveillance, Postmarketing; Registries; Vertebrates: endocrinology
• ISSN: 0021-972X; 1945-7197
• DOI: 10.1210/jc.2004-2513

Context: Small clinical trials of GH treatment of idiopathic short stature (ISS) show variable efficacy. Objective: The study was an analysis of a large GH registry for efficacy and safety of GH treatment of ISS. There was also a comparison with a specific clinical trial. Design: Up to 7 yr of GH treatment of ISS was evaluated for efficacy and safety in the National Cooperative Growth Study (NCGS). Setting: The NCGS study was conducted at Genentech, Inc. and included 47,226 patients. Patients: The ISS group included maximum stimulated GH 10 ng/ml or more and/or a report of ISS by investigator (n = 8018; all included for safety). Cohort 1 (n = 2520) was similar to the clinical trial, cohort 2 (n = 283) included subjects younger than 5 yr of age, and cohort 3 (n = 940) was pubertal at GH start. Intervention: GH, approximately 0.30 mg/kg·wk, was given. Main Outcome Measures: These included growth velocities and height sd (HtSDS). Results: Mean first-year growth velocities in cohorts 1, 2, and 3 increased 4.6, 3.9, and 4.4 cm/yr over pretreatment, respectively. Measures included: baseline mean HtSDS, −2.9, −3.2, and −2.8; mean HtSDS at 1 yr, −2.4, −2.3, and −2.3, respectively. Mean HtSDS after 7 yr in cohorts 1 (n = 303) and 2 (n = 85) and 5 yr in cohort 3 (n = 58) were: −1.2, −1.0, and −1.5, respectively. Cohort 3 shorter treatment time was due to advanced baseline age (mean 13.8 yr) and puberty. Mean HtSDS gain in cohort 1 was comparable with the clinical trial. No new safety signals specific to the NCGS ISS population were observed. Conclusion: ISS patients in the GH registry demonstrate a significant increase in HtSDS with the safety profile similar to GH-deficient patients. Results were similar to the clinical trial.