Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: A randomized, double-blind, placebo-controlled trial

Werber Leschek, Ellen, Rose, Susan R., Yanovski, Jack A., Troendle, James F., Quigley, Charmian A., Chipman, John J., Crowe, Brenda J., Ross, Judith L., Cassorla, Fernando G., Blum, Werner F., Cutler, Gordon B. Jr, Baron, Jeffrey, NICHD-Eli Lilly and Company Growth Hormone Collaborative Group and Leong, Gary M. (2004) Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: A randomized, double-blind, placebo-controlled trial. Journal of Clinical Endocrinology and Metabolism, 89 7: 3140-3148. doi:10.1210/jc.2003-031457


Author Werber Leschek, Ellen
Rose, Susan R.
Yanovski, Jack A.
Troendle, James F.
Quigley, Charmian A.
Chipman, John J.
Crowe, Brenda J.
Ross, Judith L.
Cassorla, Fernando G.
Blum, Werner F.
Cutler, Gordon B. Jr
Baron, Jeffrey
NICHD-Eli Lilly and Company Growth Hormone Collaborative Group
Leong, Gary M.
Title Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: A randomized, double-blind, placebo-controlled trial
Journal name Journal of Clinical Endocrinology and Metabolism   Check publisher's open access policy
ISSN 0021-972X
1945-7197
Publication date 2004-07-01
Sub-type Article (original research)
DOI 10.1210/jc.2003-031457
Volume 89
Issue 7
Start page 3140
End page 3148
Total pages 9
Place of publication Bethesda, MD, U.S.A.
Publisher Endocrine Society
Language eng
Subject 1103 Clinical Sciences
1114 Paediatrics and Reproductive Medicine
Formatted abstract
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.
Keyword Endocrinology & Metabolism
National cooperative growth
GH-deficient patients
Final height
Normal girls
Therapy
Puberty
Insulin
Boys
Secretion
Disadvantage
Q-Index Code C1
Additional Notes The NICHD-Eli Lilly and Co. Growth Hormone Collaborative Group includes the following investigators, who made important contributions to this study: Donna B. Bernstein, R.N.; Joy C. Jones, R.N.; Suvimol C. Hill, M.D.; Gale Heavner, R.N. [U.S. Public Health Service (USPHS) Commissioned Officer]; Mark A. Bach, M.D.; Helén L. Dichek, M.D.; Jill D. Jacobson, M.D.; Karen Oerter Klein, M.D.; Barbara L. Linder, M.D.; Gary M. Leong, M.D., Ph.D.; Saul Malozowski, M.D.; Gabriela Marin, M.D.; Susan B. Nunez, M.D.; Kristen Roberts, M.S.; Denise C. Scott, M.D.; Elena P. Shavrikova, M.S.; Pamela M. Thomas, M.D.; and Karen K. Winer, M.D.

Document type: Journal Article
Sub-type: Article (original research)
Collections: Excellence in Research Australia (ERA) - Collection
School of Medicine Publications
 
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Created: Thu, 20 Sep 2007, 04:47:56 EST