Growth hormone deficiency
Growth hormone deficiency (GHD), also known as dwarfism or pituitary dwarfism is a condition caused by insufficient amounts of growth hormone in the body.
GHD can result from various causes, including pituitary gland disorders, genetic mutations, or other medical conditions. Children with GHD may have stunted growth, while adults may experience changes in body composition, reduced bone density, and altered metabolism.
The predominant features of the adult growth hormone deficiency syndrome may vary between patients of different ages and ages of onset of GHD. Evidence from clinical trials and long-term observational studies have informed our ability to understand the unique considerations regarding the risks and benefits of daily growth hormone replacement therapy (GHRT) and specific dosing and monitoring strategies for these patient subgroups. High rates of nonadherence with daily GHRT present a challenge to achieving optimal treatment outcomes and long-acting growth hormone (LAGH) formulations have been developed with the promise of improving treatment adherence resulting in improved therapeutic outcomes. While existing data from short-term studies have demonstrated noninferiority of efficacy and safety of LAGH compared to daily GHRT, long-term studies are needed to assess the full spectrum of outcomes of interest and long-term safety considerations specific to patients in adolescence, adulthood, and the elderly GHD population. Since each LAGH formulation has a unique pharmacodynamic and pharmacokinetic profile optimal dosing and monitoring strategies will need to be developed to allow for the provision of individualized patient treatment 1).
Individuals surviving cancer and brain tumors may experience growth hormone (GH) deficiency as a result of tumor growth, surgical resection and/or radiotherapy involving the hypothalamic-pituitary region. Given the pro-mitogenic and anti-apoptotic properties of GH and insulin-like growth factor-I, the safety of GH replacement in this population has raised hypothetical safety concerns that have been debated for decades. Data from multicenter studies with extended follow-up have generally not found significant associations between GH replacement and cancer recurrence or mortality from cancer among childhood cancer survivors. Potential associations with secondary neoplasms, especially solid tumors, have been reported, although this risk appears to decline with longer follow-up. Data from survivors of pediatric or adult cancers who are treated with GH during adulthood are scarce, and the risk versus benefit profile of GH replacement of this population remains unclear. Studies pertaining to the safety of GH replacement in individuals treated for nonmalignant brain tumors, including craniopharyngioma and Non-Functioning Pituitary Neuroendocrine Tumor, have generally been reassuring with regards to the risk of tumor recurrence 2).
Clinical features
Children with GHD have abnormally short stature with normal body proportions.