Long-Term Results with Growth Hormone Therapy in Idiopathic Hypopituitarism

Bernasconi, Sergio; Arrigo, Teresa; Wasniewsk, Malgorzata; Ghizzoni, Lucia; Ruggeri, C; Pasquale, Giuseppe Di; Vottero, Alessandra; Luca, Filippo De

doi:10.1159/000053206

Cited by 10 publications

(6 citation statements)

References 31 publications

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“…Early data of final height of GHD patients receiving GH therapy were disappointing, with mean adult height reported to be approximately –1.3 standard deviation (SD) below the population mean [1,2,3,4,5,6,7,8] and –0.4 to –0.6 SD below the mid-parental height (MPH) [4,9,10]. In more recent studies, mean adult height was appropriate for MPH in patients with GHD [11,12,13].…”

Section: Introductionmentioning

confidence: 99%

Response to Growth Hormone Treatment in Isolated Growth Hormone Deficiency versus Multiple Pituitary Hormone Deficiency

2011

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Background: Growth hormone (GH) therapy successfully increases height prognosis in children with GH deficiency (GHD); however, adult height data are still limited. Aim: This study investigated near-adult height (NAH) in patients with idiopathic GHD (i.e. those with a GH peak <10 µg/l with no organic pathology) divided into two groups: isolated GHD and multiple pituitary hormone deficiency (MPHD). Methods: All patients were registered in the Pfizer International Growth Study Database (KIGS). Median (10th to 90th percentile) values are given and measurements were expressed as standard deviation scores (SDS). Parental-adjusted height was determined. Results: GH therapy was started at a median age of 9.2 (range 4.9–12) years in patients with isolated GHD (n = 1,619, 60% males) and at 7.7 (range 2.8–12.2) years in those with MPHD (n = 554, 65% males; p < 0.001) at a median dose of 0.20 mg/kg/week. Height SDS at onset of therapy was –3.1 (range –4.5 to –2.1) and –3.8 (range –5.7 to –2.3), respectively (p < 0.001). The maximum GH peak and insulin-like growth factor I SDS were significantly (p < 0.05) lower in patients with MPHD than in those with isolated GHD. Both groups showed a significant (p < 0.05) increase in height SDS at 1 year that continued until the onset of puberty. Parental-adjusted height at the start of puberty was –0.1 (range –1.6 to 1.1) in patients with isolated GHD and –0.4 (range –1.9 to 1.2) in those with MPHD. Parental-adjusted NAH SDS in patients with isolated GHD was 0.0 (range –1.5 to 1.2) and slightly, but significantly, higher than NAH (–0.3, range –2.1 to 1.2; p < 0.001) in patients with MPHD. In patients with isolated GHD, total change in height SDS while receiving GH therapy was 1.6 (range 0.5–3.2), and the change in height SDS at puberty was 0.1 (range –0.7 to 1). The respective values were 2.6 (range 0.9–4.6) and 0.2 (range –1 to 1.3) in patients with MPHD. Parental-adjusted NAH was slightly lower in girls than in boys with isolated GHD, but no gender difference was observed in patients with MPHD. Multivariate analysis in patients with GHD and MPHD showed that higher birth weight, taller parents, greater height at onset, first-year responsiveness, and predicted height velocity were the most important predictors of NAH. Conclusions: 89% of patients with isolated GHD and 81% of those with MPHD reached an NAH within their genetic potential while receiving GH therapy. Most of the height gain occurred during prepubertal years.

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Section: Introductionmentioning

confidence: 99%

Response to Growth Hormone Treatment in Isolated Growth Hormone Deficiency versus Multiple Pituitary Hormone Deficiency

2011

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“…Recombinant human growth hormone (rhGH) has been successfully synthesized and massively produced since the human GH gene was obtained using bio-engineering recombinant DNA techniques in the early 1980s and was transduced into prokaryotic or eukaryotic cells [1]. For more than 20 years, rhGH lyophilized powder for injection has been widely used in the clinical treatment of GHD and achieved satisfying therapeutic results [2][3][4][5][6][7][8][9][10]. However, rhGH lyophilized powder for injection requires to be dissolved before injection, which is complex and may cause a risk to the children, thus affecting drug compliance and efficacy [11].…”

Section: Introductionmentioning

confidence: 99%

Clinical evaluation of recombinant human growth hormone injection in children with growth hormone deficiency

Hou

Luo

et al. 2009

Front. Med. China

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Recombinant human growth hormone (rhGH) has been widely used in the clinical treatment of growth hormone deficiency. To simplify the injection process and increase drug compliance, application of the GH injection has become a new treatment plan in recent years. The purpose of the current study was to evaluate the efficacy and safety of rhGH injection for the treatment of growth hormone deficiency (GHD) in children in China. In a nationwide, noncomparative, prospective, randomized, open trial, 31 children with confirmed complete GHD received subcutaneous injection of rhGH at 0.25 mg/kg$wk (0.107 IU/kg$d). The injection was given daily and the total weekly amount was separated into 6-7 injections. The patients were followed up at 3-month intervals and the treatment duration was 12 months. The height (HT), annual growth velocity (GV), mean height standard deviation score (HT SDS), blood serum insulin-like growth factor I (IGF-I), insulin-like growth factor binding protein 3 (IGFBP-3), and bone maturity before and after treatment were compared, and the safety of the treatment was analyzed. The mean HT, GV, and HT SDS were increased from 109.0AE14 cm, 2.7AE0.9 cm/yr, and -4.62 AE1.46 at baseline to 121.8AE13.4 cm, 12.9AE3.3 cm/yr, and -2.47AE1.86 after 12 months of treatment, respectively (P < 0.001). At the same time, blood IGF-I and IGFBP-3 were increased significantly [41.27AE64.43 μg/L vs 159.21AE167.92 μg/L and 1540.00AE1325.11 mg/L vs 3533.93AE1413.82 mg/L, respectively (P < 0.001)]. The bone age assessments performed 6 and 12 months after the treatment showed that no advanced bone maturation was noted. No serious adverse events occurred during the treatment, and the drug-related adverse events were mainly decreased thyroid function. We conclude that rhGH injection is a safe and effective drug for treatment of growth hormone deficiency in children.

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“…Patients with growth hormone deficiency (GHD) who receive treatment with biosynthetic growth hormone (GH) have markedly improved actual or near-final height (NFH) outcomes, with an average final height approximating –1.3 standard deviations (SD) below the mean reported in more than 1,400 patients from different registries and trials [1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]. Yet despite the availability of modern GH therapy, long-term studies still show that the majority of patients fail to achieve their genetic target heights.…”

Section: Introductionmentioning

confidence: 99%

The KIGS Experience with the Addition of Gonadotropin-Releasing Hormone Agonists to Growth Hormone (GH) Treatment of Children with Idiopathic GH Deficiency

Reiter

Lindberg²,

Ranke³

et al. 2003

Horm Res Paediatr

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Although recombinant techniques have enabled the production of limitless amounts of human growth hormone (GH), and clinical methods for diagnosis and treatment have been greatly enhanced, the mean final heights of children with idiopathic GH deficiency (IGHD) treated with GH remain in the range of –1.3 standard deviation scores (SDS) below normal height. One of the methods used to increase height outcomes is to delay the onset and progression of puberty to allow for a longer ‘pre-pubertal’ growth phase. We reviewed the KIGS (Pharmacia International Growth Database) data of patients with IGHD who had been treated with gonadotropin-releasing hormone agonists (GnRHa) in order to see if a greater gain in height could be achieved by altering the tempo of pubertal maturation. Near-final height data were analysed in 39 adolescents (out of a total of 249) who had received GH + GnRHa therapy and were compared with similar data from 1,893 patients with IGHD treated with GH alone. The total change in height SDS in boys who received GH alone was +1.6, in contrast to +1.1 in GH + GnRHa-treated boys; the total change in height SDS in girls who received GH alone was +1.4 in contrast to +1.1 in girls treated with GH + GnRHa. The near final height SDS in girls treated with GH + GnRHa was 1.0 below the mid-parental target height (MPH), whereas there was only a –0.5 SDS difference in girls treated with GH. Approximation to the MPH did not differ in boys between the two treatment groups. These data suggest that the attainment of a substantial height SDS by manipulating the tempo of puberty is limited, but that optimizing growth during the pre-pubertal phase is a more important factor.

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Long-Term Results with Growth Hormone Therapy in Idiopathic Hypopituitarism

Cited by 10 publications

References 31 publications

Response to Growth Hormone Treatment in Isolated Growth Hormone Deficiency versus Multiple Pituitary Hormone Deficiency

Response to Growth Hormone Treatment in Isolated Growth Hormone Deficiency versus Multiple Pituitary Hormone Deficiency

Clinical evaluation of recombinant human growth hormone injection in children with growth hormone deficiency

The KIGS Experience with the Addition of Gonadotropin-Releasing Hormone Agonists to Growth Hormone (GH) Treatment of Children with Idiopathic GH Deficiency

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