Three‐year data from a comparative study with recombinant human growth hormone in the treatment of short stature in young children with intrauterine growth retardation

Abstract

Growth acceleration and bone maturation were studied for 3 y in 69 children with severe short stature and a history of intrauterine growth retardation (IUGR), to determine the effect of treatment with recombinant human growth hormone (r‐hGH). The patients were enrolled in an open, multicentre trial and were randomly allocated to either the treated group (Group 1) 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, during 3 y and the children in Group 2 started the study with a 1‐y observation 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‐GHdeficient, 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 body mass index SDS was ‐1.4. After 1 y of treatment, linear HV in Group 1 increased in comparison with the pre‐treatment period (from 5.7 ± 2.0 to 10.1 ± 1.7cm/y;p < 0:001)and with the firstyear of observation in Group2(p < 0:001). Increased HV was sustained during the second and third year of treatment and was significantly higher than at baseline. A similar growth pattern was seen during the 3y of GH treatment 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 treatment was negatively correlated with growth velocity at baseline. Bone age remained retarded but increased with a mean of almost 4 y after 3y of treatment in both groups. Even at a dose that is three times the replacement dose treatment with r‐hGH was well tolerated. From these results, we conclude that r‐hGH treatment over 3 y can induce sustained catch‐up growth in young children with severe short stature and a history of IUGR. Long‐term studies are needed to assess ultimate effects on final height.