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

被引：8

作者：

Counts, Debra R. ^{[1
]}

Silverman, Lawrence A. ^{[2
]}

Rajicic, Natasa ^{[3
]}

Geffner, Mitchell E. ^{[6
]}

Newfield, Ron S. ^{[7
]}

Thornton, Paul ^{[8
]}

Carakushansky, Mauri ^{[9
]}

Escobar, Oscar ^{[10
]}

Rapaport, Robert ^{[4
]}

Levitsky, Lynne ^{[11
]}

Rotenstein, Deborah ^{[11
]}

Hey-Hadavi, Judith ^{[3
]}

Wajnrajch, Michael P. ^{[3
,5
]}

机构：

[1] Univ Maryland, Sch Med, Baltimore, MD 21201 USA

[2] Goryeb Childrens Hosp, Morristown, NJ USA

[3] Pfizer Inc, New York, NY USA

[4] Icahn Sch Med Mt Sinai, New York, NY 10029 USA

[5] NYU, New York, NY USA

[6] USC, Keck Sch Med, Los Angeles, CA USA

[7] Univ Calif San Diego, Sch Med, La Jolla, CA 92093 USA

[8] Cook Childrens Med Ctr, Ft Worth, TX USA

[9] Nemours Childrens Hosp, Orlando, FL USA

[10] Univ Pittsburgh, Sch Med, Boston, MA USA

[11] Harvard Univ, Sch Med, Boston, MA USA

来源：

HORMONE RESEARCH IN PAEDIATRICS | 2015年 / 84卷 / 02期

关键词：

Cost effectiveness; Clinical trial; Growth hormone; Dose of growth hormone; Height prediction model; Short stature; PREPUBERTAL CHILDREN; GH TREATMENT; INTERNATIONAL GROWTH; FINAL HEIGHT; THERAPY; AGE; RESPONSIVENESS; VALIDATION; PREDICTION; PERIOD;

D O I：

10.1159/000381642

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 (R) versus a widely used ISS dose of Genotropin (R). 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. (C) 2015 S. Karger AG, Basel

引用

页码：79 / 87

页数：9

共 1 条

[1] 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
Counts, D. R.
Silverman, L. A.
Geffner, M. E.
Rajicic, N.
Hey-Hadavi, J.
Thornton, P. S.
Wajnrajch, M. P.
HORMONE RESEARCH IN PAEDIATRICS, 2013, 80 (04): : 242 - 251

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