Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a randomized, double-blind, placebo-controlled trial.
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 < or = -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.
Leschek
Ellen Werber
EW
Developmental Endocrinology Branch, National Institute of Child Health and Human Development/NIH, Building 10, Room 10N262, 10 Center Drive, MSC 1862, Bethesda, MD 20892-1862, USA.
Rose
Susan R
SR
Yanovski
Jack A
JA
Troendle
James F
JF
Quigley
Charmian A
CA
Chipman
John J
JJ
Crowe
Brenda J
BJ
Ross
Judith L
JL
Cassorla
Fernando G
FG
Blum
Werner F
WF
Cutler
Gordon B
GB
Baron
Jeffrey
J
D000293:Adolescent; D001827:Body Height / Q000187:drug effects*; D002648:Child; D004311:Double-Blind Method; D005260:Female; D006130:Growth Disorders / Q000188:drug therapy* / Q000473:pathology; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D010919:Placebos; D011627:Puberty; D016896:Treatment Outcome
D010919:Placebos; D019382:Human Growth Hormone