Additionally, although new, teduglutide and
rhGH have shown promising increases in intestinal absorption qualities in SBS (Carter et al., 2017; Goulet, Dabbas-Tyan et al., 2010; Guo et al., 2012).
In the late 1990s, randomized controlled studies confirmed the positive effect of
rhGH therapy on the course of PWS.
In this study, we aimed to evaluate response to GH treatment in patients who had a definite diagnosis of hypochondroplasia by clinical, radiological and genetic examination, who also met the criteria for growth hormone deficiency and who received
rhGH replacement therapy.
The patient was started on recombinant human growth hormone (
rhGH) therapy at age 7.75 years with an increase in her annualized growth velocity from 4.4 cm/yr pre-rhGH to 9.3 cm/yr after year 1 (increase in height SDS from -2.5 to -1.1) and then to 10.5 cm/yr at the end of year 2 (height SDS of 0) (Figure 1).
To determine the type of GHD, we also performed a genetic analysis that allowed us to diagnose type IA GHD; therefore, substitution therapy was initiated with
rhGH. The literature reports a genotype/phenotype correlation in patients with IGHD [20]; children with type IA IGHD may have birth short length and neonatal hypoglycaemia along with undetectable GH concentrations with severe growth failure in the first 6 months.
Parisi et al., "Supra-physiological
rhGH administration induces gender-related differences in the hypothalamus-pituitary-thyroid (HPT) axis in healthy individuals," Journal of Endocrinological Investigation, vol.
Venous blood collection logistics may limit the number of antidoping controls for those substances, like
rhGH, that can be detected only in blood.
The International Consensus Conference on
rhGH replacement therapy, (53) suggests that severe GHD should be treated.
Also, "as most of the cancers associated with acromegaly and/or high GH/IGF-I levels are cancers seen predominantly in adults, long-term studies are needed to determine if treatment with
rhGH [recombinant human GH] in childhood is associated with an increased risk for the common adult cancers."
The study is expected to enroll up to 136 naive to treatment, pre-pubertal children with GHD and will include a 3:1 randomization of 3.5 mg/kg VRS-317 semi-monthly to daily
rhGH at the highest approved dose on the labels of Genotropin(R) and Norditropin(R) 34 ug/kg/day.