scholarly journals One-Year Data from a Long-Term Phase IV Study of Recombinant Human Growth Hormone in Short Children Born Small for Gestational Age

2014 ◽  
Vol 4 (1-2) ◽  
pp. 1-13 ◽  
Author(s):  
Hans-Peter Schwarz ◽  
Dorota Birkholz-Walerzak ◽  
Mieczyslaw Szalecki ◽  
Mieczyslaw Walczak ◽  
Corina Galesanu ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Human Growth Hormone ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Short Children ◽  
One Year ◽  
Phase Iv

scholarly journals Two-Year Data from a Long-Term Phase IV Study of Recombinant Human Growth Hormone in Short Children Born Small for Gestational Age

2016 ◽  
Vol 33 (3) ◽  
pp. 423-434 ◽  
Author(s):  
Hans-Peter Schwarz ◽  
Mieczysław Walczak ◽  
Dorota Birkholz-Walerzak ◽  
Mieczyslaw Szalecki ◽  
Michaela Nanu ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Human Growth Hormone ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Short Children ◽  
Phase Iv

scholarly journals Efficacy and safety of the recombinant human growth hormone in short children born small for gestational age

Medicine ◽  
2021 ◽  
Vol 100 (30) ◽  
pp. e26711
Author(s):  
Su Jin Kim ◽  
Min-Sun Kim ◽  
Sung Yoon Cho ◽  
Byung-Kyu Suh ◽  
Cheol Woo Ko ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Human Growth Hormone ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Efficacy And Safety ◽  
Short Children

Growth and metabolic effects of long-term recombinant human growth hormone (rhGH) treatment in short children born small for gestational age: GH-RAST study

2020 ◽  
Vol 0 (0) ◽  
Author(s):  
José I. Labarta ◽  
Antonio de Arriba ◽  
Marta Ferrer ◽  
Marisa Loranca ◽  
José María Martos ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Human Growth Hormone ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Duration Of Treatment ◽  
Short Children ◽  
Height Gain ◽  
Rhgh Treatment

AbstractObjectivesTo study the efficacy and influence on metabolism of recombinant human growth hormone (rhGH) treatment in short children born small for gestational age (SGA).MethodsRetrospective, observational, multicenter study in 305 short children born SGA, treated with rhGH during a mean ± SD of 5.03 ± 1.73 years at a mean ± SD dose of 37 ± 8 μg/kg/day. Auxological and metabolic assessment including glucose and lipids profile were collected.ResultsMean ± SD age at the start of treatment was 7.11 ± 2.78 years. Height and weight improved significantly until the end of treatment from mean −2.72 (CI95%: −2.81 to −2.63) standard deviation score (SDS) to −1.16 (CI95%: −1.44 to −0.88) SDS and from −1.62 (CI95%: −1.69 to −1.55) SDS to −0.94 (CI95%: −1.14 to −0.74) SDS respectively. Mean height gain was 1.27 (CI95%: 0.99–1.54) SDS. Prepubertal patients showed higher height gain than pubertal children (mean [CI95%] = 1.44 [CI95%: 1.14–1.74] vs. 0.73 [CI95%: 0.22–1.24], p=0.02). Height gain SDS during treatment negatively correlated with chronological age (CA) and bone age (BA) delay and positively correlated with duration of treatment, height gain during first year of treatment, years on prepubertal treatment and height SDS from target height (TH). Glucose, insulin, and triglycerides increased significantly but remained within the normal range. Total and LDL-cholesterol decreased significantly, and HDL-cholesterol remained unchanged.ConclusionsrhGH treatment in short SGA children effectively normalized height in most of the patients and showed a safe metabolic profile. Children who benefit the most are those with greater height SDS distance from TH, BA delay, longer duration of treatment and prepubertal treatment initiation.

scholarly journals Practical tools to identify short children born small-for-gestational-age eligible for rhGH treatment according to Italian regulation

2019 ◽  
Vol 45 (1) ◽  
Author(s):  
Gianluca Tornese ◽  
Flavia Pricci ◽  
Maria Chiara Pellegrin ◽  
Marika Villa ◽  
Daniela Rotondi ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
National Registry ◽  
Hormone Deficiency ◽  
Number Of Children ◽  
Short Children ◽  
Rhgh Treatment

Abstract Recombinant human growth hormone (rhGH) is an approved and effective treatment for short children born small for gestational age (SGA). Prevalence of children eligible for treatment as SGA is reported to be 1:1800. The latest data from the National Registry of Growth Hormone therapy (RNAOC) showed that the number of children treated with SGA indication is still small (prevalence 0.37/100,000) and these children are significantly less reported than those treated for growth hormone deficiency (GHD), although GHD prevalence is 1:4000–1:10,000. This means that many short children born SGA are still not properly identified, and therefore not treated with rhGH, or misdiagnosed as GHD. This article provides some practical tools for the identification of children eligible for rhGH treatment.

Treatment of Short Stature in Aggrecan Deficient Patients with Recombinant Human Growth Hormone: One-Year Response

2021 ◽  
Author(s):  
Gajanthan Muthuvel ◽  
Andrew Dauber ◽  
Eirene Alexandrou ◽  
Leah Tyzinski ◽  
Melissa Andrew ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Short Stature ◽  
Human Growth Hormone ◽  
Linear Growth ◽  
Recombinant Human Growth Hormone ◽  
Human Growth ◽  
Skeletal Maturation ◽  
Height Velocity ◽  
Heterozygous Mutation ◽  
One Year

Abstract Context Patients with aggrecan (ACAN) deficiency present with dominantly inherited short stature, often with advanced skeletal maturation and premature growth cessation. There is a paucity of information on the effects of growth-promoting interventions. Objective The aim of this study was to evaluate the efficacy and safety of recombinant human growth hormone (rhGH) therapy on linear growth in children with ACAN deficiency. Design and Setting Open-label, single-arm, prospective study at Cincinnati Children’s Hospital Medical Center. Patients Ten treatment-naïve patients were recruited. Inclusion criteria were: a confirmed heterozygous mutation in ACAN, age ≥ 2 years, pre-pubertal, bone age (BA) ≥ chronological age (CA), and normal IGF-I concentration. Intervention Treatment with rhGH (50 mcg/kg/day) over one year. Main Outcome Measure(s) Main outcomes measured were height velocity (HV) and change in (Δ) height SD (HtSDS). Results Ten patients (six females) were enrolled with median CA of 5.6 yrs (range 2.4 to 9.7). Baseline median HtSDS was -2.5 (range -4.3 to -1.1). Median baseline BA was 6.9 yrs (range 2.5 to 10.0), with median BA/CA of 1.2 (range 0.9 to 1.5). Median pre-treatment HV was 5.2 cm/y (range 3.8 to 7.1), increased to 8.3 cm/y (range 7.3 to 11.2) after one year of therapy (p=0.004). Median ΔHtSDS after one year was +0.62 (range +0.35 to +1.39) (p=0.002). Skeletal maturation did not advance inappropriately (median Δ BA/CA -0.1, p=0.09). No adverse events related to rhGH were observed. Conclusion Treatment with rhGH improved linear growth in a cohort of patients with short stature due to ACAN deficiency.

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