A 4-year, open-label, multicenter, randomized trial of genotropin® growth hormone in patients with idiopathic short stature

Analysis of 4-year data comparing efficacy, efficiency, and safety between an individualized, target-driven regimen and standard dosing

Debra R. Counts, Lawrence Silverman, Natasa Rajicic, Mitchell E. Geffner, Ron S. Newfield, Paul Thornton, Mauri Carakushansky, Oscar Escobar, Robert Rapaport, Lynne Levitsky, Deborah Rotenstein, Judith Hey-Hadavi, Michael P. Wajnrajch

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Background/Aims: Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment. Methods: This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin® versus a widely used ISS dose of Genotropin®. Subjects were prepubertal, had a bone age of 3-10 years for males and 3-9 years for females, were naive to GH treatment, and had a height standard deviation score (Ht SDS) of -3 to -2.25, a height velocity <25th percentile for their bone age, and peak stimulated GH >10 ng/ml. After the first 2 years, the individualized-dosing group was further randomized to either 0.18 or 0.24 mg/kg/week. Results: At 4 years, subjects in all treatment regimens achieved similar average height gains of +1.3 SDS; however, the individualized dosing regimen utilized less GH to achieve an equivalent height gain. Conclusion: Individualized, formula-based GH dosing, followed by a dose reduction after 2 years, provides a more cost-effective growth improvement in patients with ISS than currently employed weight-based regimens.

Original languageEnglish (US)
Pages (from-to)79-87
Number of pages9
JournalHormone Research in Paediatrics
Volume84
Issue number2
DOIs
StatePublished - Aug 27 2015

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Human Growth Hormone
Growth Hormone
Multicenter Studies
Safety
Weights and Measures
Therapeutics
Hormones
Costs and Cost Analysis
Bone and Bones
Growth

All Science Journal Classification (ASJC) codes

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

Cite this

Counts, Debra R. ; Silverman, Lawrence ; Rajicic, Natasa ; Geffner, Mitchell E. ; Newfield, Ron S. ; Thornton, Paul ; Carakushansky, Mauri ; Escobar, Oscar ; Rapaport, Robert ; Levitsky, Lynne ; Rotenstein, Deborah ; Hey-Hadavi, Judith ; Wajnrajch, Michael P. / A 4-year, open-label, multicenter, randomized trial of genotropin® growth hormone in patients with idiopathic short stature : Analysis of 4-year data comparing efficacy, efficiency, and safety between an individualized, target-driven regimen and standard dosing. In: Hormone Research in Paediatrics. 2015 ; Vol. 84, No. 2. pp. 79-87.
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abstract = "Background/Aims: Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment. Methods: This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin{\circledR} versus a widely used ISS dose of Genotropin{\circledR}. Subjects were prepubertal, had a bone age of 3-10 years for males and 3-9 years for females, were naive to GH treatment, and had a height standard deviation score (Ht SDS) of -3 to -2.25, a height velocity <25th percentile for their bone age, and peak stimulated GH >10 ng/ml. After the first 2 years, the individualized-dosing group was further randomized to either 0.18 or 0.24 mg/kg/week. Results: At 4 years, subjects in all treatment regimens achieved similar average height gains of +1.3 SDS; however, the individualized dosing regimen utilized less GH to achieve an equivalent height gain. Conclusion: Individualized, formula-based GH dosing, followed by a dose reduction after 2 years, provides a more cost-effective growth improvement in patients with ISS than currently employed weight-based regimens.",
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A 4-year, open-label, multicenter, randomized trial of genotropin® growth hormone in patients with idiopathic short stature : Analysis of 4-year data comparing efficacy, efficiency, and safety between an individualized, target-driven regimen and standard dosing. / Counts, Debra R.; Silverman, Lawrence; Rajicic, Natasa; Geffner, Mitchell E.; Newfield, Ron S.; Thornton, Paul; Carakushansky, Mauri; Escobar, Oscar; Rapaport, Robert; Levitsky, Lynne; Rotenstein, Deborah; Hey-Hadavi, Judith; Wajnrajch, Michael P.

In: Hormone Research in Paediatrics, Vol. 84, No. 2, 27.08.2015, p. 79-87.

Research output: Contribution to journalArticle

TY - JOUR

T1 - A 4-year, open-label, multicenter, randomized trial of genotropin® growth hormone in patients with idiopathic short stature

T2 - Analysis of 4-year data comparing efficacy, efficiency, and safety between an individualized, target-driven regimen and standard dosing

AU - Counts, Debra R.

AU - Silverman, Lawrence

AU - Rajicic, Natasa

AU - Geffner, Mitchell E.

AU - Newfield, Ron S.

AU - Thornton, Paul

AU - Carakushansky, Mauri

AU - Escobar, Oscar

AU - Rapaport, Robert

AU - Levitsky, Lynne

AU - Rotenstein, Deborah

AU - Hey-Hadavi, Judith

AU - Wajnrajch, Michael P.

PY - 2015/8/27

Y1 - 2015/8/27

N2 - Background/Aims: Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment. Methods: This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin® versus a widely used ISS dose of Genotropin®. Subjects were prepubertal, had a bone age of 3-10 years for males and 3-9 years for females, were naive to GH treatment, and had a height standard deviation score (Ht SDS) of -3 to -2.25, a height velocity <25th percentile for their bone age, and peak stimulated GH >10 ng/ml. After the first 2 years, the individualized-dosing group was further randomized to either 0.18 or 0.24 mg/kg/week. Results: At 4 years, subjects in all treatment regimens achieved similar average height gains of +1.3 SDS; however, the individualized dosing regimen utilized less GH to achieve an equivalent height gain. Conclusion: Individualized, formula-based GH dosing, followed by a dose reduction after 2 years, provides a more cost-effective growth improvement in patients with ISS than currently employed weight-based regimens.

AB - Background/Aims: Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment. Methods: This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin® versus a widely used ISS dose of Genotropin®. Subjects were prepubertal, had a bone age of 3-10 years for males and 3-9 years for females, were naive to GH treatment, and had a height standard deviation score (Ht SDS) of -3 to -2.25, a height velocity <25th percentile for their bone age, and peak stimulated GH >10 ng/ml. After the first 2 years, the individualized-dosing group was further randomized to either 0.18 or 0.24 mg/kg/week. Results: At 4 years, subjects in all treatment regimens achieved similar average height gains of +1.3 SDS; however, the individualized dosing regimen utilized less GH to achieve an equivalent height gain. Conclusion: Individualized, formula-based GH dosing, followed by a dose reduction after 2 years, provides a more cost-effective growth improvement in patients with ISS than currently employed weight-based regimens.

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