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Submitted on December 26, 2006
Accepted on June 18, 2007
University of North Carolina, Chapel Hill, North Carolina, 27599; Lilly Research Laboratories, Indianapolis, Indiana, 46285; The Children's Hospital, affiliated with the University of Colorado Health Sciences Center, Denver, Colorado, 80218; Connecticut Children's Medical Center, Hartford, Connecticut, 06106; Thomas Jefferson University, Philadelphia, Pennsylvania, 19107;Children's Hospital Medical Center, Seattle, WA, 98105; The Saban Research Institute of the Children's Hospital Los Angeles, Los Angeles, California, 90027; University of Arkansas for Medical Sciences, Little Rock, Arkansas, 72205; Children's Mercy Hospital, Kansas City, Missouri, 64108
* To whom correspondence should be addressed. E-mail: mld{at}med.unc.edu.
Context: Typically, growth failure in Turner syndrome (TS) begins prenatally, and height standard deviation score (SDS) declines progressively from birth.
Objective: This study aimed to determine whether GH treatment initiated before 4 yr of age in girls with TS could prevent subsequent growth failure. Secondary objectives were to identify factors associated with treatment response, to determine whether outcome could be predicted by a regression model using these factors and to assess the safety of GH treatment in this young cohort.
Design: This study was a prospective, randomized, controlled, open-label, multi-center clinical trial (‘Toddler Turner Study’; August 1999-August 2003).
Setting: The study was conducted at 11 U.S. pediatric endocrine centers.
Subjects: Eighty-eight girls with TS, aged 9 months-4 yr, were enrolled.
Intervention(s): Interventions comprised recombinant GH (50 µg/kg/day, n=45) or no treatment (n=43) for 2 yr.
Main Outcome Measure(s): The main outcome measure was baseline-to-2-yr change in height standard deviation score (SDS).
Results: Short stature was evident at baseline (mean length/height SDS = -1.6±1.0 at mean age 24.0±12.1 months). Mean height SDS increased in the GH group from -1.4±1.0 to -0.3±1.1 (1.1 SDS gain), whereas it decreased in the control group from -1.8±1.1 to -2.2±1.2 (0.5 SDS decline), resulting in a 2-yr between-group difference of 1.6±0.6 SDS (P<0.0001). The baseline variable that correlated most strongly with 2-yr height gain was the difference between mid-parental height (MPH) SDS and subjects' height SDS (r=0.32, P=0.04). Although attained height SDS at 2 yr could be predicted with good accuracy using baseline variables alone (R2=0.81, p<0.0001) prediction of 2-yr change in height SDS required inclusion of initial treatment response data (4-month or 1-yr height velocity) in the model (R2=0.54, p<0.0001). No new or unexpected safety signals associated with GH treatment were detected.
Conclusion: Early GH treatment can correct growth failure and normalize height in infants and toddlers with TS.
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  B. Birgit, H. Julius, R. Carsten, S. Maryam, F. Gabriel, K. Victoria, F. Margareta, and H. Outi Fertility Preservation in Girls with Turner Syndrome: Prognostic Signs of the Presence of Ovarian Follicles J. Clin. Endocrinol. Metab., January 1, 2009; 94(1): 74 - 80. [Abstract] [Full Text] [PDF]
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 B. E. Hjerrild, K. H. Mortensen, and C. H. Gravholt Turner syndrome and clinical treatment Br. Med. Bull., June 1, 2008; 86(1): 77 - 93. [Abstract] [Full Text] [PDF]
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