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


METHODS
Up to 7 yr of GH treatment of ISS was evaluated for efficacy and safety in the National Cooperative Growth Study (NCGS).


METHODS
The NCGS study was conducted at Genentech, Inc. and included 47,226 patients.


METHODS
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.


METHODS
GH, approximately 0.30 mg/kg.wk, was given.


METHODS
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.


CONCLUSIONS
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.
University of Arkansas for Medical Sciences, Arkansas Children's Hospital, 800 Marshall Street, Little Rock, Arkansas 72202-3591, USA. kempstephenf@uams.edu
Stephen F
SF
Kemp
Joyce
J
Kuntze
Kenneth M
KM
Attie
Thomas
T
Maneatis
S
S
Butler
James
J
Frane
Barbara
B
Lippe
D000293:Adolescent; D001827:Body Height / Q000187:drug effects*; D002648:Child; D015331:Cohort Studies; D004334:Drug Administration Schedule; D005260:Female; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000008:administration & dosage* / Q000009:adverse effects* / Q000627:therapeutic use; D006801:Humans; D008297:Male; D011358:Product Surveillance, Postmarketing; D012042:Registries
D019382:Human Growth Hormone