Endocrine Abstracts

While a number of studies have shown that treatment of children born small for gestational age (SGA) with continuously administered growth hormone (GH) induces a significant height improvement, this clinical trial approved by the local ethics committee investigated in a prospective way the growth response to different intermittent recombinant human GH (r-hGH, Saizen) treatment regimens over 4 years.

Fifty-eight severely short (height standard deviation score (HSDS) of -3.49 ± 0.67), very young (3.24 ± 0.78 years) children born SGA were randomised into two treatment regimens with r-hGH at a daily dose of 0.067 mg/kg, equal to a cumulative dose of 0.033 mg/kg.day over 4 years. Thirty patients received a single two-year treatment cycle followed by 2 years of observation (TTOO). Twenty-eight patients received two 1-year treatment cycles, in the first and third year of the four-year study period (TOTO). Both treatment regimens showed a significant and comparable increase in height velocity (HV), HV standard deviation score (HVSDS) and height SDS (HSDS) during the active treatment years. During the years off-treatment a clear decline of the auxological parameters was observed. Both treatment regimens resulted in a significant and comparable increase in HSDS at the end of the 4 year period (from -3.54 ± 0.61 to -1.99 ± 0.81 in the TTOO group and from -3.43 ± 0.74 to -2.00 ± 1.01 in the TOTO group). OGTT-induced insulin secretion was slightly but reversibly increased only in the TTOO group. The treatment was otherwise well tolerated.

These results demonstrate in a prospective way that intermittent treatment regimens have a beneficial effect on height in short children born SGA. Moreover, they show that the efficacy of 2 years r-hGH treatment given as different intermittent regimens over a total period of 4 years, with the same cumulative r-hGH dose, are comparable.