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Effect of Growth Hormone Treatment on Adult Height in Peripubertal Children with Idiopathic Short Stature: A Randomized, Double-Blind, Placebo-Controlled Trial

Developmental Endocrinology Branch (E.W.L., J.A.Y., J.B.) and Biometry and Mathematical Statistics Branch (J.F.T.), National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland 20892; Department of Endocrinology (S.R.R.), Children’s Hospital Medical Center, Cincinnati, Ohio 45229; Eli Lilly and Company (C.A.Q., J.J.C., B.J.C., W.F.B., G.B.C.), Indianapolis, Indiana 46285; Department of Pediatrics (J.L.R.), Thomas Jefferson University, Philadelphia, Pennsylvania 19107; Alfred I. duPont Hospital for Children (J.L.R.), Wilmington, Delaware 19899; and Institute of Maternal and Child Research (F.G.C.), University of Chile, Santiago, Chile

Address all correspondence and requests for reprints to: Jeffrey Baron, M.D., National Institutes of Health, Building 10, Room 10N262, 10 Center Drive, MSC 1862, Bethesda, Maryland 20892-1862. E-mail: jeffrey_baron{at}nih.gov.

GH is often used to treat children with idiopathic short stature despite the lack of definitive, long-term studies of efficacy. We performed a randomized, double-blind, placebo-controlled trial to determine the effect of GH on adult height in peripubertal children. Subjects (n = 68; 53 males and 15 females), 9–16 yr old, with marked, idiopathic short stature [height or predicted height –2.5 SD score (SDS)] received either GH (0.074 mg/kg) or placebo sc three times per week until they were near adult height. At study termination, adult height measurements were available for 33 patients after mean treatment duration of 4.4 yr. Adult height was greater in the GH-treated group (–1.81 ± 0.11 SDS, least squares mean ± SEM) than in the placebo-treated group (–2.32 ± 0.17 SDS) by 0.51 SDS (3.7 cm; P < 0.02; 95% confidence interval, 0.10–0.92 SDS). A similar GH effect was demonstrated in terms of adult height SDS minus baseline height SDS and adult height SDS minus baseline predicted height SDS. Modified intent-to-treat analysis in 62 patients treated for at least 6 months indicated a similar GH effect on last observed height SDS (0.52 SDS; 3.8 cm; P < 0.001; 95% confidence interval, 0.22–0.82 SDS) and no important dropout bias. In conclusion, GH treatment increases adult height in peripubertal children with marked idiopathic short stature.

This work was supported in part by Eli Lilly and Co.. J.A.Y. and J.B. are Commissioned Officers in the USPHS.

Abbreviations: ANCOVA, Analysis of covariance; CI, confidence interval; SDS, SD score.

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