Growth hormone treatment of early growth failure in toddlers with turner syndrome: A randomized, controlled, multicenter trial

Marsha L. Davenport, Brenda J. Crowe, Sharon H. Travers, Karen Rubin, Judith L. Ross, Patricia Y. Fechner, Daniel F. Gunther, Chunhua Liu, Mitchell E. Geffner, Kathryn Thrailkill, Carol Huseman, Anthony J. Zagar, Charmian A. Quigley

Research output: Contribution to journalArticlepeer-review

104 Scopus citations

Abstract

Context: Typically, growth failure in Turner syndrome (TS) begins prenatally, and height SD 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, openlabel, multicenter clinical trial (Toddler Turner Study, August 1999 to August 2003). Setting: The study was conducted at 11 U.S. pediatric endocrine centers. Subjects: Eighty-eight girls with TS, aged 9 months to 4 yr, were enrolled. Interventions: Interventions comprised recombinant GH (50 μg/kg·d; n = 45) or no treatment (n = 43) for 2 yr. Main Outcome Measure: The main outcome measure was baselineto-2-yr change in height 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 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.

Original languageEnglish
Pages (from-to)3406-3416
Number of pages11
JournalJournal of Clinical Endocrinology and Metabolism
Volume92
Issue number9
DOIs
StatePublished - Sep 2007

Bibliographical note

Funding Information:
This work was supported by Eli Lilly and Company (Indianapolis, IN). Portions of this work were conducted through and supported by the National Institutes of Health-funded General Clinical Research Center facilities at the University of Washington and Children's Hospital and Regional Medical Center (RR00037) and the University of North Carolina at Chapel Hill, North Carolina (RR00046). In compliance with the Uniform Requirements for Manuscripts, established by the International Committee of Medical Journal Editors, the sponsor of this study did not impose any impediment, directly or indirectly, on the publication of the study's results. The authors acknowledge the independent medical writing assistance provided by ProScribe Medical Communications ( www.proscribe.com.au ), funded by Eli Lilly and Company. ProScribe's services complied with international guidelines for Good Publication Practice.

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Endocrinology
  • Clinical Biochemistry
  • Biochemistry, medical

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