Growth hormone therapy with three dosage regimens in children with idiopathic short stature. European Study Group Participating Investigators

• Published in: The Journal of pediatrics Vol. 132; no. 3 Pt 1; pp. 455 - 460
• Main Authors: Rekers-Mombarg, L T, Massa, G G, Wit, J M, Matranga, A M, Buckler, J M, Butenandt, O, Chaussain, J L, Frisch, H, Leiberman, E, Yturriaga, R, Aarskog, D, Chatelain, P G, Colle, M, Dacou-Voutetakis, C, Delemarre-van de Waal, H A, Girard, F, Gosen, J J, Irle, U, Jansen, M, Jean, R, Job, J C, Kaar, M L, Kollemann, F, Lenko, H L, Waelkens, J J
• Format: Journal Article
• Language: English
• Published: United States 01.03.1998
• Subjects: Body Height - drug effects; Child; Dose-Response Relationship, Drug; Female; Fetal Growth Retardation; Growth - drug effects; Growth Disorders - drug therapy; Growth Disorders - physiopathology; Growth Hormone - administration & dosage; Humans; Male; Regression Analysis
• ISSN: 0022-3476

In children with idiopathic short stature (ISS) we studied the growth-promoting effect at 4 years of recombinant human growth hormone (rhGH) therapy in three dose regimens and evaluated whether increasing the dosage after the first year could prevent a decline in height velocity (HV). Included were 223 patients who were treated with subcutaneous administrations of rhGH 6 days per week. They were randomized to three groups: 3 IU/m2 body surface/day, 4.5 IU/m2/day, and 3 IU/m2/day during the first year and 4.5 IU/m2/day thereafter, corresponding with dosages of 0.2 and 0.3 mg/kg body weight/week, respectively. Growth was compared with a standard of 229 untreated children with ISS [ISS standard]. During the first year of treatment HV almost doubled and was higher with 4.5 IU/m2 than with 3 IU/m2. In the second year HV no longer differed among the groups, but increasing the dosage slowed the rate of the fall of HV. During 4 years of therapy the height SD score for age increased by a mean (SD) of 2.5 (1.0) [ISS standards], or 1.2 (0.7) (British standards), bone age increased by 4.8 (1.3) years, and predicted adult height SD score increased by 1.5 (0.7). After 4 years the results of the group with 4.5 IU/m2 were slightly better than those of the other groups. When dropouts were included in the analysis (assuming a stable height SD score after discontinuation of rhGH therapy), height gain was still significant. During 4 years of rhGH therapy, growth and final height prognosis improved, slightly more with 4.5 IU/m2 than with 3 IU/m2 or 3 to 4.5 IU/m2. However, bone age advanced on average 4.8 years during this period; therefore, any effect on final height will probably be modest.