A four-year, open-label, multi-center, randomized, two-arm study of Genotropin® in patients with idiopathic short stature

Comparison of an individualized, target-driven treatment regimen to standard dosing of Genotropin® - Analysis of two-year data

D. R. Counts, Lawrence Silverman, M. E. Geffner, N. Rajicic, J. Hey-Hadavi, P. S. Thornton, M. P. Wajnrajch

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Background: Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS). Objective: To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing. Methods: A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin ® GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin® (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml. Results: The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS. Conclusion: The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.

Original languageEnglish (US)
Pages (from-to)242-251
Number of pages10
JournalHormone Research in Paediatrics
Volume80
Issue number4
DOIs
StatePublished - Oct 1 2013

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Human Growth Hormone
Growth Hormone
Therapeutics
Growth
Reference Values
Hormones
Safety
Weights and Measures
Bone and Bones

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Endocrinology, Diabetes and Metabolism
  • Endocrinology

Cite this

@article{1ffd82925d5f45568909ca750de07fc5,
title = "A four-year, open-label, multi-center, randomized, two-arm study of Genotropin{\circledR} in patients with idiopathic short stature: Comparison of an individualized, target-driven treatment regimen to standard dosing of Genotropin{\circledR} - Analysis of two-year data",
abstract = "Background: Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS). Objective: To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing. Methods: A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin {\circledR} GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin{\circledR} (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml. Results: The majority (83{\%}) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS. Conclusion: The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.",
author = "Counts, {D. R.} and Lawrence Silverman and Geffner, {M. E.} and N. Rajicic and J. Hey-Hadavi and Thornton, {P. S.} and Wajnrajch, {M. P.}",
year = "2013",
month = "10",
day = "1",
doi = "10.1159/000354126",
language = "English (US)",
volume = "80",
pages = "242--251",
journal = "Hormone Research in Paediatrics",
issn = "1663-2818",
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TY - JOUR

T1 - A four-year, open-label, multi-center, randomized, two-arm study of Genotropin® in patients with idiopathic short stature

T2 - Comparison of an individualized, target-driven treatment regimen to standard dosing of Genotropin® - Analysis of two-year data

AU - Counts, D. R.

AU - Silverman, Lawrence

AU - Geffner, M. E.

AU - Rajicic, N.

AU - Hey-Hadavi, J.

AU - Thornton, P. S.

AU - Wajnrajch, M. P.

PY - 2013/10/1

Y1 - 2013/10/1

N2 - Background: Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS). Objective: To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing. Methods: A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin ® GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin® (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml. Results: The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS. Conclusion: The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.

AB - Background: Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS). Objective: To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing. Methods: A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin ® GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin® (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml. Results: The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS. Conclusion: The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.

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U2 - 10.1159/000354126

DO - 10.1159/000354126

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VL - 80

SP - 242

EP - 251

JO - Hormone Research in Paediatrics

JF - Hormone Research in Paediatrics

SN - 1663-2818

IS - 4

ER -