Adult height in children with growth hormone deficiency: a randomized, controlled, growth hormone dose-response trial

To investigate the effect of 2 growth hormone (GH) doses on adult height (AH) in GH deficiency (GHD).

A multicenter, randomized, controlled dose-response trial compared attained AH minus target height (TH) between children receiving 0.7 mg/m(2)/day biosynthetic GH (approx. 0.025 mg/kg/day) or 1.4 mg/m(2)/day (approx. 0.050 mg/kg/day). The patients enrolled in the trial were 20 'naïve' GHD children (had not received GH before) and 15 'transfer' GHD children (already on GH for at least 1 year).

In the naïve group, the mean +/- SD AH minus TH was -5.3 +/- 6.1 and -2.2 +/- 6.9 cm in patients on 0.7 and 1.4 mg/m(2)/day, respectively (mean +/- SE difference 3.1 +/- 2.9; p = 0.3). In the transfer group, the mean +/- SD AH minus TH was -4.4 +/- 6.4 and +0.6 +/- 7.0 cm in patients on 0.7 and 1.4 mg/m(2)/day, respectively (mean +/- SE difference 5.0 +/- 3.5; p = 0.17). Spontaneous puberty started 1.1 years earlier in children on 1.4 compared to 0.7 mg/m(2)/day. Induction of puberty was more often delayed in transfer children on 0.7 than on 1.4 mg/m(2)/day.

In our GHD patients, AH was 4-5 cm less than TH in patients on 0.7 mg/m(2)/day GH, while it was 0-2 cm less in patients on 1.4 mg/m(2)/day GH, but this difference did not reach statistical significance, probably due to limited numbers of patients, considerable variability in the growth response and earlier spontaneous puberty and pubertal induction in the children on 1.4 mg/m(2)/day.