Growth hormone treatment in children with short stature born small for gestational age: 5-year results of a randomized, double-blind, dose-response trial

The growth-promoting effect of continuous GH treatment was evaluated over 5 yr in 79 children with short stature (height so score, less than - 1.88) b arn small for gestational age (SGA; birth length sn score, less than -1.88) , Patients were randomly and, blindly assigned to I of 2 GH dosage groups ( 3 vs. 6 IU/m(2) body surface day). GN:deficiency was not an exclusion crite rium. After 5 yr of GH treatment almost every child had reached a height we ll within the normal range for healthy Dutch children and in the range of t heir target height sn score. Only in children who remained prepubertal duri ng the study period was the 5-yr increase in height so score (HSDS) for chr onalogical age significantly higher in the study group receiving 6 com-pare d to 3 IU GH/m(2).day. Remarkably, the 5-yr increment in HSDS for chronolog ical age was not related to spontaneous GH secretion, maximum GB levels aft er provocation, or baseline insulin-like growth factor I levels. GH treatme nt was associated with an acceleration of bone maturation regardless of the GH dose given. The HSDS for bone age and predicted adult height increased significantly. GH treatment was well tolerated. In conclusion, our 5-yr data show that long term continuous GH treatment at a dose of 3 or 6 IU/m2 day in short children born SGA results in a normali zation of height during childhood followed by growth along the target heigh t percentile.