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J Clin Endocrinol Metab. 2005 Sep;90(9):5247-53. Epub 2005 Jul 5.

Efficacy and safety results of long-term growth hormone treatment of idiopathic short stature.

Author information

1
University of Arkansas for Medical Sciences, Arkansas Children's Hospital, 800 Marshall Street, Little Rock, Arkansas 72202-3591, USA. kempstephenf@uams.edu

Abstract

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.

PMID:
15998780
DOI:
10.1210/jc.2004-2513
[Indexed for MEDLINE]

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