Improvement in Adult Height after Growth Hormone Treatment in Adolescents with Short Stature Born Small for Gestational Age: Results of a Randomized Controlled Study

• Published in: The journal of clinical endocrinology and metabolism 2003-04, Vol.88 (4), p.1587-1593
• Main Authors: Carel, Jean-Claude, Chatelain, Pierre, Rochiccioli, Pierre, Chaussain, Jean-Louis
• Format: Article
• Language: English
• Subjects: Adolescent; Biological and medical sciences; Body Height; Child; Female; Hormones. Endocrine system; Human Growth Hormone - adverse effects; Human Growth Hormone - therapeutic use; Humans; Infant, Newborn; Infant, Small for Gestational Age; Insulin-Like Growth Factor I - analysis; Male; Medical sciences; Pharmacology. Drug treatments; Time Factors; Treatment Outcome
• ISSN: 0021-972X; 1945-7197
• DOI: 10.1210/jc.2002-021123

The efficacy of GH for increasing adult height (AH) in short adolescents born small for gestational age (SGA) is unclear, due to the lack of long-term controlled trials. A total of 168 short children born SGA (age, 10.5 yr for girls and 12.5 yr for boys) were randomly assigned to receive either 0.067 mg/kg·d GH until attainment of AH or no treatment. In this per-protocol analysis, 91 of 102 patients in the treated group and 33 of 47 patients in the control group were followed to AH. Mean height at inclusion was −3.2 sd score (SDS). Treatment duration was 2.7 ± 0.6 yr. AH was −2.7 ± 0.9 and −2.1 ± 1.0 SDS in the control and treated groups, respectively (P < 0.005). The groups differed by 0.6 SDS units (95% confidence interval, 0.2–0.9). Height gain was 0.5 ± 0.8 and 1.1 ± 0.9 SDS in the control and treated groups, respectively (P = 0.002). Multivariate analyses confirmed the independent effects of treatment (0.6 SDS) and treatment duration (0.4 SDS/yr). All potential biases would tend to decrease the estimate of the treatment effect. Treatment tolerance was excellent. We concluded that the potential for spontaneous catch-up in short adolescents born SGA is limited. GH treatment increases AH by at least 0.6 SDS in this population.