Comparison of response to 2-years’ growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies

Abstract

BackgroundFew studies have compared the response to growth hormone (GH) treatment between indications such as isolated growth hormone deficiency (IGHD), born small for gestational age (SGA), idiopathic short stature (ISS), and multiple pituitary hormone deficiency (MPHD). The aim of this analysis of data, collected from two large ongoing observational outcome studies, was to evaluate growth and insulin-like growth factor-I (IGF-I) response data for children of short stature with IGHD, MPHD, SGA, or ISS following two years of treatment with the recombinant GH product Norditropin® (Novo Nordisk A/S, Bagsværd, Denmark).MethodsAnalysis of auxologic data from two ongoing prospective observational studies, NordiNet® International Outcomes Study (NordiNet® IOS) and NovoNet®/American Norditropin® Studies: Web-enabled Research (ANSWER) Program®.Results4,582 children aged <18 years were included: IGHD, n = 3,298; SGA, n = 678; ISS, n = 334; and MPHD, n = 272. After two years’ GH treatment, change in height standard deviation score (SDS) was +1.03 in SGA and +0.84 in ISS vs. +0.97 in IGHD (p = 0.047; p < 0.001 vs. IGHD, respectively). Height gain was comparable between IGHD and MPHD. In pre-pubertal children vs. total population, height SDS change after two years was: IGHD, +1.24 vs. +0.97; SGA, +1.17 vs. +1.03; ISS, +1.04 vs. +0.84; and MPHD, +1.16 vs. +0.99 (all p < 0.001).ConclusionsAfter two years’ GH treatment, change in height SDS was greater in SGA and less in ISS, compared with IGHD; the discrepancy in responses may be due to the disease nature or confounders (i.e. age). Height SDS increase was greatest in pre-pubertal children, supporting early treatment initiation to optimize growth outcomes.

Baseline Characteristics and Gender Differences in Prepubertal Children Treated with Growth Hormone in Europe, USA, and Japan: 25 Years' KIGS® Experience (1987-2012) and Review

“…Despite such discrepancies, it is worth remarking that GH levels were very similar among the individual diagnostic groups and across the 3 regions ( Table 2 ). While the height pattern in prepubertal males and females in this study was comparable to that of children in the Nordinet ® study [20] , the girls with IGHD in the Australian OZGROW ® study [13] were younger, but not shorter ( Table 4 ).…”

Prepubertal Children with Growth Hormone Deficiency Treated for Four Years with Growth Hormone Experience Dose-Dependent Increase in Height, but Not in the Rate of Puberty Initiation

“…>−2 SDS). Mean change in IGF-1 SDS remained within the reference range (±2 SDS) after 2 years at a mean dose of 38 μg/kg/day [20]. IGF-1 SDS data beyond the initial 24 months of rhGH treatment are unavailable for the present prospective study.…”

Growth Hormone Treatment for Short Stature in the USA, Germany and France: 15 Years of Surveillance in the Genetics and Neuroendocrinology of Short-Stature International Study (GeNeSIS)

“…Other analyses have also shown differences in response to GH treatment across diagnostic groups [31, 32]. For example, Lee et al [31] showed that change in height SDS was greater in children born SGA and smaller in those with idiopathic short stature when compared with children with idiopathic GHD.…”

“…In addition, final height of girls with Turner syndrome is positively influenced by height at GH initiation, the GH dose and age at onset of puberty, and negatively influenced by age at treatment initiation [29, 30]. Other analyses have also shown differences in response to GH treatment across diagnostic groups [31, 32]. For example, Lee et al [31] showed that change in height SDS was greater in children born SGA and smaller in those with idiopathic short stature when compared with children with idiopathic GHD.…”