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Early differentiation between good and poor response to growth hormone therapy in short children born small for gestational age (SGA) to improve the outcome of poor responders

  • Heike Jung , Christof Land , Werner F. Blum and Eckhard Schöenau EMAIL logo
Published/Copyright: October 23, 2013

Abstract

Objective: The aim of this study was to examine height-gain response in relation to predicted good or poor response during first-year low or high growth hormone (GH) dose in short prepubertal children born small for gestational age.

Patients and methods: The OPTIMA (Optimization of GH Treatment in Short Children Born Small for Gestational Age Based on a Growth Prediction Model) randomised study evaluated 12-month height standard deviation score (SDS) changes in patients receiving GH dose: fixed high (FH; 0.067 mg/kg/day) or 0.035 mg/kg/day individually adjusted (IA) after 3 months according to the Cologne early growth prediction.

Results: Predicted 12-month height SDS gain was <0.75 for 21/89 FH-dose patients, considered poor responders; 11/21 reached a 12-month height SDS gain of ≥0.75. In IA-dose poor responders, increasing GH dose at 3 months maintained mean height velocity (HV), with 73.7% reaching a 12-month height SDS gain of ≥0.75 vs. 73.8% in IA-dose good responders who continued on low GH dose, where mean HV decreased after the initial 3-month period.

Conclusion: GH dose increase at 3 months in patients with predicted poor response maintained catch-up growth. Even when on FH dose, some patients did not achieve a good response.


Corresponding author: Eckhard Schöenau, Pediatric Endocrinology Department, Children’s Hospital, University of Cologne, Joseph-Stelzmann-Strasse 9, D-50924 Cologne, Germany, E-mail:

Acknowledgments

The authors are very grateful for the cooperation of all of the investigators involved in the OPTIMA study. The authors also thank Dr. Peter Bates, Cambridge Medical Writing Services, UK, for his help in the preparation of the manuscript, and Claudia Nicolay, Lilly Deutschland, for her support in the statistical analysis.

Conflict of interest statement

Author’s conflict of interest disclosure: E.S. is a member of Medical Research advisory boards for, and E.S. and C.L. have received consulting fees and travel expenses from, Eli Lilly and Company. H.J. and W.F.B. are employees and stockholders of Eli Lilly and Company.

Funding source: The OPTIMA study was funded by Eli Lilly and Company, Indianapolis, IN, USA.

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Received: 2013-4-19
Accepted: 2013-9-9
Published Online: 2013-10-23
Published in Print: 2014-03-01

©2014 by Walter de Gruyter Berlin Boston

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