title: 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-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 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 +/- 1.7 cm/y; p < 0.001) and with the first year of observation in Group 2 (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 3 y 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 3 y 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.
authors.affiliation: Hôpitaux Robert Debré, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Fjellestad-Paulsen
authors.forename: P
authors.initials: P
authors.lastname: Czernichow
authors.forename: R
authors.initials: R
authors.lastname: Brauner
authors.forename: M
authors.initials: M
authors.lastname: Bost
authors.forename: M
authors.initials: M
authors.lastname: Colle
authors.forename: J Y
authors.initials: JY
authors.lastname: Lebouc
authors.forename: M
authors.initials: M
authors.lastname: Lecornu
authors.forename: B
authors.initials: B
authors.lastname: Leheup
authors.forename: J M
authors.initials: JM
authors.lastname: Limal
authors.forename: M C
authors.initials: MC
authors.lastname: Raux
authors.forename: J E
authors.initials: JE
authors.lastname: Toublanc
authors.forename: R
authors.initials: R
authors.lastname: Rappaport
mesh_terms: D000293:Adolescent; D001827:Body Height; D015992:Body Mass Index; D002648:Child; D002675:Child, Preschool; D005260:Female; D005317:Fetal Growth Retardation / Q000188:drug therapy*; D005500:Follow-Up Studies; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011627:Puberty; D016896:Treatment Outcome
chemical_list: D019382:Human Growth Hormone