3-YEAR DATA FROM A COMPARATIVE-STUDY WITH RECOMBINANT HUMAN GROWTH-HORMONE IN THE TREATMENT OF SHORT STATURE IN YOUNG-CHILDREN WITH INTRAUTERINE GROWTH-RETARDATION

Growth acceleration and bone maturation were studied for 3 y in 69 chi ldren with severe short stature and a history of intrauterine growth r etardation (IUGR), to determine the effect of treatment with recombina nt human growth hormone (r-hGH). The patients were enrolled in an open , multicentre trial and were randomly allocated to either the treated group (Group I)or the control group (Group 2). The children in Group 1 were treated daily with 0.2 IU/kg/body weight (0.067 mg/kg) s.c., dur ing 3 y and the children in Group 2 started the study with a 1-y obser vation period followed by a 3-y treatment period. At birth, their mean weight standard deviation score (SDS) was -2.5 and their mean length SDS -3.5. At baseline, the patients were prepubertal, non-GH deficient , with no known dysmorphic features. Mean age was 4.5 y, bone age was 3.3 y, height SDS was -3.4, height velocity (HV) SDS was -1.6, and bod y mass index SDS was -1.4. After I y of treatment, linear HV in Group 1 increased in comparison with the pre-treatment period (from 5.7 +/- 2.0 to 10.1 +/- 1.7 cm/y; p < 0.001) and with the first year of observ ation in Group 2 (p < 0.001). Increased KV was sustained during the se cond and third year of treatment and was significantly higher than at baseline. A similar growth pattern was seen during the 3 y of CH treat ment in Group 2. Mean height SDS for chronological age increased by 2. 0 +/- 0.7 in the two groups after 3 y of treatment. HV after 1 y of tr eatment was negatively correlated with growth velocity at baseline. Bo ne age remained retarded but increased with a mean of almost 4 y after 3 y of treatment in both groups. Even at a dose that is three times t he replacement dose treatment with r-hGH was well tolerated. From thes e results, we conclude that r-hGH treatment over 3 y can induce sustai ned catch-up growth in young children with severe shea stature and a h istory of IUGR. Long-term studies are needed to assess ultimate effect s on final height.