Delving into the Latest Updates on Recombinant GH(Hamad General Hospital) with Synapse

Overview

Basic Info

Drug Type

Hormone

Synonyms-

Target-

Mechanism-

Therapeutic Areas

Other Diseases

Active Indication

Idiopathic short stature

Inactive Indication-

Originator Organization

King Hamad General Hospital

Active Organization

King Hamad General Hospital

Inactive Organization-

Drug Highest PhaseClinical

First Approval Date-

Regulation-

Open-label, single-arm, prospective study over 3 years recruiting 10 treatment-naïve patients with heterozygous mutations in ACAN, age ≥2 years, prepubertal, and normal IGF-I concentration. Patients were treated with rhGH (initially, 50 mcg/kg/day). Main outcomes were change in (Δ) height SD score (HtSDS) and height velocity (HV).

Results:

Ten patients (6 females) enrolled with median chronological age (CA) of 5.6 years (range, 2.4-9.7). Baseline median HtSDS, HV, and bone age/CA were −2.5 (range, −4.3 to −1.1), 5.2 cm/year (range, 3.8 to 7.1), and 1.2 (range, 0.9 to 1.5), respectively. The cumulative median ΔHtSDS over 3 years was +1.21 (range, +0.82 to +1.94). Median HV increased to 8.3 cm/year (range, 7.3-11.2), 7.7 cm/year (range, 5.9-8.8), and 6.8 cm/year (range, 4.9-8.6) during years 1, 2, and 3, respectively. The median Δ predicated adult height was +6.8 cm over 3 years. Four female subjects entered puberty; nevertheless, median Δbone age/CA was −0.1. No adverse events related to rhGH were observed.

Conclusion:

Linear growth improved in a cohort of ACAN-deficient patients treated with rhGH, albeit somewhat attenuated in older participants who entered puberty. Longitudinal follow-up is needed to assess the long-term efficacy of rhGH and adult height outcome.

01 Oct 2024·Translational Pediatrics

Effect of growth hormone to spinal growth and recombinant human growth hormone to scoliosis

Review

Author: Peng, Danxia ; Liu, Qing ; Xu, Xuan ; Wen, Jie ; Li, Yanyan ; Luo, Cheng ; Wu, Qiong ; Liu, Shuping ; Han, Shu

Growth hormone (GH) plays a key role in human growth and development. In addition to promoting height growth, GH affects bone metabolism, bone size, and bone mineral density (BMD) in children and adolescents by affecting bone formation and resorption. Among them, the effect of GH on spinal growth has been widely concerned. Scoliosis is a three-dimensional structural spinal deformity characterized by lateral curvature of one or more segments of the spine accompanied by vertebral rotation and sagittal imbalance. For children with growth hormone deficiency (GHD), whether GH supplementation leads to scoliosis is still controversial. In recent years, numerous scholars have conducted extensive research to investigate the correlation between recombinant human GH replacement therapy and scoliosis, yielding divergent findings with some even presenting contradictory results. This study aims to investigate the impact of GH on spinal growth and explore the association between recombinant human GH replacement therapy and scoliosis by comprehensively reviewing the effects of GH and insulin-like growth factors 1 (IGF-1) on bone metabolism, bone mass, as well as examining the consequences of GHD on bone health. Additionally, we aim to access the influence of recombinant human GH replacement therapy on adolescent idiopathic scoliosis (AIS).

14 Aug 2024·JCEM Case Reports

Growth Hormone Deficiency in an Adolescent With Pseudohypoparathyroidism Type 1B

Article

Author: Sasidharan Pillai, Sabitha ; Reyes, Monica ; Topor, Lisa Swartz ; Jüppner, Harald

Abstract:

We report growth hormone (GH) deficiency due to presumed GH releasing hormone (GHRH) resistance in an adolescent with pseudohypoparathyroidism type 1B (PHP1B) due to paternal uniparental disomy of chromosome 20 (patUPD20). A male patient aged 11 years 10 months with obesity and mild developmental delay was found to have hypocalcemia, hyperphosphatemia, and an elevated parathyroid hormone level. History included muscle cramps and leg pain with activity. Examination showed round facies, short stature, and obesity. He was in puberty and bone age was advanced by > 2 years. Detailed genetic workup, including nucleotide sequence analysis of GNAS exons 1-13 and STX16, methylation-sensitive multiplex ligation-dependent probe amplification and analysis of several microsatellite markers for chromosome 20, established the diagnosis of PHP1B due to patUPD20. Muscle cramps and hypocalcemia resolved with calcium carbonate, ergocalciferol, and calcitriol treatment. He was short with linear growth deceleration at around age 13 years. Peak GH concentration was insufficient following stimulation testing. Growth velocity improved with human GH treatment. Although rare, resistance to GHRH can occur in PHP1B and patients with this disorder should be evaluated for GH insufficiency if they present with short stature and reduced growth velocity. Treatment with recombinant human GH may improve growth velocity in such patients.

100 Deals associated with Recombinant GH(Hamad General Hospital)

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