Growth hormone treatment in non-growth hormone-deficient children

Abstract

Until 1985 growth hormone (GH) was obtained from pituitary extracts, and was available in limited amounts only to treat severe growth hormone deficiency (GHD). With the availability of unlimited quantities of GH obtained from recombinant DNA technology, researchers started to explore new modalities to treat GHD children, as well as to treat a number of other non-GHD conditions. Although with some differences between different countries, GH treatment is indicated in children with Turner syndrome, chronic renal insufficiency, Prader-Willi syndrome, deletions/mutations of the SHOX gene, as well as in short children born small for gestational age and with idiopathic short stature. Available data from controlled trials indicate that GH treatment increases adult height in patients with Turner syndrome, in patients with chronic renal insufficiency, and in short children born small for gestational age. Patients with SHOX deficiency seem to respond to treatment similarly to Turner syndrome. GH treatment in children with idiopathic short stature produces a modest mean increase in adult height but the response in the individual patient is unpredictable. Uncontrolled studies indicate that GH treatment may be beneficial also in children with Noonan syndrome. In patients with Prader-Willi syndrome GH treatment normalizes growth and improves body composition and cognitive function. In any indication the response to GH seems correlated to the dose and the duration of treatment. GH treatment is generally safe with no major adverse effects being recorded in any condition.

Challenges in the Diagnosis and Management of Growth Hormone Deficiency in India

“…Other genes known to be associated with SS include PTPN11 , SOS1 (NS), FGFR3 (achondroplasia and hypochondroplasia), SHOX (SHOX-D), NPR2 , aggrecan, and PAPPA2 [54–56]. SHOX deficiency is the first indication being approved for GH treatment that requires genetic testing [20]. The availability of genetic testing for SS syndrome is limited outside metropolitan cities and is expensive.…”

Growth hormone (GH) peak in stimulation tests and the severity of IGF-I deficiency before treatment as the predictors of GH therapy effectiveness in children with short stature

“…According to the very recent paper of Loche et al [5], the mean increase of adult height in patients with ISS during the rhGH therapy is modest and the individual response to treatment is unpredictable. In our study, growth response to treatment was significantly better in patients with severe IGFD than in those with normal IGF-I levels and almost independent of GH peak in stimulation tests.…”

“…In the patients with normal GH secretion and excluded GH insensitivity, in whom the diagnosis of idiopathic short stature (ISS) seems most appropriate [1], the rhGH therapy is still a matter of controversy [2][3][4]. Lower efficacy of the rhGH therapy in children with ISS than in those with GHD has been reported, however with a high variability of growth response among treated patients [2,[5][6][7]. A few years ago, GHD has been defined as secondary insulin-like growth factor-I (IGF-I) deficiency (IGFD) [8][9].…”

Craniofacial morphology and dental maturity in children with reduced somatic growth of different aetiology and the effect of growth hormone treatment

“…Precise terminology and the ability to distinguish normal from abnormal growth have never been more important than at present because of the increasing tend to administer growth hormone (GH) to children without any signs of growth hormone deficiency (GHD), whose heights are within or below the low normal range [ 5 , 6 ]. Treatment of children and adolescents with idiopathic short stature.…”