Height velocity targets from the national cooperative growth study for first-year growth hormone responses in short

Bert Bakker1, James Frane, Henry Anhalt

  • 1Genentech, Inc., 1 DNA Way, mailstop 454 B, South San Francisco, California 94080, USA. bakker.bert@gene.com

Insights

This study establishes age- and gender-specific growth targets for children receiving growth hormone (GH) therapy. These benchmarks help assess treatment effectiveness and identify potential issues in growth hormone deficiency and short stature.

Area of Science:

  • Pediatric Endocrinology
  • Growth Hormone Therapy
  • Clinical Pediatrics

Background:

  • Growth hormone (GH) has been used for over 40 years to treat short stature.
  • Established criteria for defining satisfactory GH responsiveness targets are lacking.
  • This gap hinders optimal treatment assessment for various pediatric growth conditions.

Purpose of the Study:

  • To derive age-specific targets for GH responsiveness in prepubertal children.
  • To analyze first-year height velocity (HV) data from Genentech's National Cooperative Growth Study.
  • To establish benchmarks for idiopathic GH deficiency (GHD), organic GHD, idiopathic short stature, and Turner syndrome.

Main Methods:

  • Utilized data from the National Cooperative Growth Study.
  • Constructed first-year HV response curves to standard daily GH doses.
  • Analyzed naive-to-treatment prepubertal children across defined diagnostic categories.

Main Results:

  • Presented HV plots stratified by age at baseline for each condition and gender.
  • Mean -2 standard deviation (SD) HV plots closely approximated pretreatment HV.
  • Established reference curves for evaluating GH treatment outcomes.

Conclusions:

  • Proposed age- and gender-specific targets to aid clinicians in assessing first-year growth response.
  • Defined a "poor" response as HV below the mean -1 SD on these plots.
  • These curves can guide GH dose adjustments, compliance assessments, and diagnostic reviews.
Abstract

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