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The Journal of Clinical Endocrinology & Metabolism Vol. 88, No. 4 1587-1593
Copyright © 2003 by The Endocrine Society

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

Jean-Claude Carel, Pierre Chatelain, Pierre Rochiccioli and Jean-Louis Chaussain

Department of Pediatric Endocrinology and Institut National de la Santé et de la Recherche Médicale U561 (J.-C.C., J.-L.C.), Groupe Hospitalier Cochin-Saint Vincent de Paul and Faculté Cochin-Université Paris V, 75014 Paris, France; Department of Pediatric Endocrinology (P.C.), Hôpital Debrousse, 69005 Lyon, France; and Department of Pediatric Endocrinology (P.R.), Hôpital des Enfants, 31059 Toulouse, France

Address all correspondence and requests for reprints to: Prof. Jean-Claude Carel, Pediatric Endocrinology and Institut National de la Santé et de la Recherche Médicale U561, Groupe Hospitalier Cochin-Saint Vincent de Paul, 82 av Denfert Rochereau, 75014 Paris, France. E-mail: carel{at}cochin.inserm.fr.

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.

This study was funded by Sanofi-Synthélabo (Paris, France).

Abbreviations: AH, Adult height; CI, confidence interval; SDS, SD score; SGA, small for gestational age.




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