Recombinant human growth hormone for children born small for gestational age: meta-analysis confirms the consistent

R Crabbé1, M von Holtey, P Engrand

  • 1Debiopharm SA, Lausanne, Switzerland.

Insights

Higher doses of recombinant human growth hormone (r-hGH) improve growth in short children born small for gestational age (SGA). Treatment with Serono

Area of Science:

  • Pediatric Endocrinology
  • Growth Hormone Therapy
  • Biopharmaceuticals

Background:

  • Optimal recombinant human growth hormone (r-hGH) dosing for short children born small for gestational age (SGA) remains under investigation.
  • Children born SGA often experience growth deficits requiring therapeutic intervention.

Purpose of the Study:

  • To determine the dose-response relationship of r-hGH in short children born SGA.
  • To evaluate the efficacy of a specific r-hGH formulation (Serono) at a standard dose.

Main Methods:

  • Meta-analysis of clinical trials involving r-hGH treatment in SGA children.
  • Development of a mathematical model to describe the dose-response relationship.
  • Estimation and comparison of growth response (height-SDS gain) to different r-hGH doses and formulations.

Main Results:

  • A clear dose-response relationship was established, with higher r-hGH doses yielding greater height-SDS gain.
  • The higher dose (0.067 mg/kg/day) showed a mean difference of 0.48 SDS gain compared to the lower dose (0.033 mg/kg/day).
  • The Serono r-hGH formulation at 0.033 mg/kg/day demonstrated an estimated height-SDS gain of 1.2, consistent with other formulations.

Conclusions:

  • A significant dose-response relationship exists for r-hGH in treating short SGA children.
  • Treatment with Serono r-hGH formulation at 0.033 mg/kg/day is expected to yield a meaningful therapeutic outcome.
Abstract

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