Adult height of prepubertal short children born small for gestational age treated with GH.
OBJECTIVE
Human GH (hGH) treatment leads to catch-up growth in children with short stature born small for gestational age (SGA). However, long-term efficacy and safety results in this patient group remain scarce. The present study assessed the efficacy and safety of late childhood treatment with biosynthetic hGH (Humatrope) in a group of short children born SGA (height <-2 standard deviation scores (SDS)).


METHODS
Patients in this open-label, Phase III, multicenter study received a daily hGH dose of 0.067 mg/kg for 2 years, and then received no treatment for the following 2 years. After the fourth year on study, patients whose height had decreased more than 0.5 SDS but who still showed growth potential based on bone age were allowed to resume treatment until they reached adult height.


METHODS
Height gain SDS was assessed for 11 girls and 24 boys (mean age+/-s.d. 9.6+/-0.9 years) at the end of the 2 years of hGH treatment, during the subsequent 2-year off-treatment period, and upon reaching adult height.


RESULTS
At the end of the initial 2-year treatment period, 83% of patients had reached a height within the normal range, with a mean increase in height SDS vs baseline of 1.3+/-0.3 (P <0.001). Adult heights (n = 20) were within the normal range for 50% of patients, and mean height gain from baseline was statistically significant (0.7+/-0.8 SDS, P <0.001). Fasting glucose and glycosylated hemoglobin levels were not significantly modified during treatment.


CONCLUSIONS
High-dose hGH treatment for a minimum of 2 years in short children born SGA was well tolerated and resulted in a significant increase in adolescent and adult height.
Rosilio
Myriam
M
Lilly Research Laboratories, Eli Lilly and Company, Suresnes, France. rosilio_myriam@lilly.com
Carel
Jean-Claude
JC
Ecosse
Emmanuel
E
Chaussainon
Jean-Louis
JL
D001786:Blood Glucose / Q000378:metabolism; D001827:Body Height / Q000187:drug effects*; D001846:Bone Development / Q000187:drug effects; D002648:Child; D005260:Female; D006442:Glycated Hemoglobin / Q000378:metabolism; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007231:Infant, Newborn; D007236:Infant, Small for Gestational Age / Q000254:growth & development* / Q000378:metabolism; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D016014:Linear Models; D008297:Male; D010290:Parents
D001786:Blood Glucose; D006442:Glycated Hemoglobin; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I