Recombinant human growth hormone plus recombinant human insulin-like growth factor-1 coadministration therapy in short children with low insulin-like growth factor-1 and growth hormone sufficiency: Results from a randomized, multicenter, open-label, parallel-group, active treatment-controlled trial

Philippe F. Backeljauw, Bradley S. Miller, Pascale Dutailly, Aude Houchard, Elizabeth Lawson, Daniel E. Hale, Barry Reiner, Mark A. Sperling

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh)GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score ≤-2.0 and IGF-1 SD score ≤-1.0 for age and sex, and with stimulated GH ≥10 ng/ml for age and sex, were randomized to receive (all doses in μg/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and Δ height SD score were measured. Results: The first-year HV (modified intention-to-treat population) was 9.3 ± 1.7 cm/year (group A), 10.1 ± 1.3 cm/year (group B), 9.7 ± 2.5 cm/year (group C) and 11.2 ± 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, coadministration of rhGH/rhIGF-1 (45/150 μg/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies.

Original languageEnglish (US)
Pages (from-to)268-279
Number of pages12
JournalHormone Research in Paediatrics
Volume83
Issue number4
DOIs
StatePublished - May 28 2015

Fingerprint

Human Growth Hormone
Somatomedins
Growth Hormone
Therapeutics
Safety
Growth
Population

All Science Journal Classification (ASJC) codes

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

Cite this

@article{2f1538f33f904319b1db452e6bae1b6f,
title = "Recombinant human growth hormone plus recombinant human insulin-like growth factor-1 coadministration therapy in short children with low insulin-like growth factor-1 and growth hormone sufficiency: Results from a randomized, multicenter, open-label, parallel-group, active treatment-controlled trial",
abstract = "Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh)GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score ≤-2.0 and IGF-1 SD score ≤-1.0 for age and sex, and with stimulated GH ≥10 ng/ml for age and sex, were randomized to receive (all doses in μg/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and Δ height SD score were measured. Results: The first-year HV (modified intention-to-treat population) was 9.3 ± 1.7 cm/year (group A), 10.1 ± 1.3 cm/year (group B), 9.7 ± 2.5 cm/year (group C) and 11.2 ± 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, coadministration of rhGH/rhIGF-1 (45/150 μg/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies.",
author = "Backeljauw, {Philippe F.} and Miller, {Bradley S.} and Pascale Dutailly and Aude Houchard and Elizabeth Lawson and Hale, {Daniel E.} and Barry Reiner and Sperling, {Mark A.}",
year = "2015",
month = "5",
day = "28",
doi = "10.1159/000371799",
language = "English (US)",
volume = "83",
pages = "268--279",
journal = "Hormone Research in Paediatrics",
issn = "1663-2818",
publisher = "S. Karger AG",
number = "4",

}

TY - JOUR

T1 - Recombinant human growth hormone plus recombinant human insulin-like growth factor-1 coadministration therapy in short children with low insulin-like growth factor-1 and growth hormone sufficiency

T2 - Results from a randomized, multicenter, open-label, parallel-group, active treatment-controlled trial

AU - Backeljauw, Philippe F.

AU - Miller, Bradley S.

AU - Dutailly, Pascale

AU - Houchard, Aude

AU - Lawson, Elizabeth

AU - Hale, Daniel E.

AU - Reiner, Barry

AU - Sperling, Mark A.

PY - 2015/5/28

Y1 - 2015/5/28

N2 - Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh)GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score ≤-2.0 and IGF-1 SD score ≤-1.0 for age and sex, and with stimulated GH ≥10 ng/ml for age and sex, were randomized to receive (all doses in μg/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and Δ height SD score were measured. Results: The first-year HV (modified intention-to-treat population) was 9.3 ± 1.7 cm/year (group A), 10.1 ± 1.3 cm/year (group B), 9.7 ± 2.5 cm/year (group C) and 11.2 ± 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, coadministration of rhGH/rhIGF-1 (45/150 μg/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies.

AB - Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh)GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score ≤-2.0 and IGF-1 SD score ≤-1.0 for age and sex, and with stimulated GH ≥10 ng/ml for age and sex, were randomized to receive (all doses in μg/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and Δ height SD score were measured. Results: The first-year HV (modified intention-to-treat population) was 9.3 ± 1.7 cm/year (group A), 10.1 ± 1.3 cm/year (group B), 9.7 ± 2.5 cm/year (group C) and 11.2 ± 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, coadministration of rhGH/rhIGF-1 (45/150 μg/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies.

UR - http://www.scopus.com/inward/record.url?scp=84929950353&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84929950353&partnerID=8YFLogxK

U2 - 10.1159/000371799

DO - 10.1159/000371799

M3 - Article

C2 - 25765099

AN - SCOPUS:84929950353

VL - 83

SP - 268

EP - 279

JO - Hormone Research in Paediatrics

JF - Hormone Research in Paediatrics

SN - 1663-2818

IS - 4

ER -