Abstract
ObjectiveTo identify factors associated with growth in children on growth hormone (GH) therapy using data from the American Norditropin Studies: Web-enabled Research (ANSWER) Program® registry.MethodsGH-naïve children with GH deficiency, multiple pituitary hormone deficiency, idiopathic short stature, Turner syndrome, or a history of small for gestational age were eligible (N = 1,002). Using a longitudinal statistical approach, predictive factors were identified in patients with GHD for change from baseline in height standard deviation score (ΔHSDS) following 2 years of treatment.ResultsGradual increases in ΔHSDS over time were observed for all diagnostic categories. Significant predictive factors of ΔHSDS, ranked by significance were: height velocity (HV) at 4 months > baseline age > baseline HSDS > baseline body mass index (BMI) SDS > baseline insulin-like growth factor I (IGF-I) SDS; gender was not significant. HV at 4 months and baseline BMI SDS were positively correlated, whereas baseline age, HSDS, and IGF-I SDS were negatively correlated with ΔHSDS.ConclusionsThese results may help guide GH therapy based on pretreatment characteristics and early growth response.
Highlights
Treatment with exogenous growth hormone (GH) has become a well-accepted therapeutic option for children with growth failure
Since the availability of recombinant human GH in 1985, a wide range of conditions associated with decreased growth, including GH deficiency (GHD), Turner syndrome (TS), Noonan syndrome (NS), children born small for gestational age (SGA), Prader-Willi syndrome (PWS), idiopathic short stature (ISS), and SHOX gene haploinsufficiency have been approved by the United States Food and Drug Administration (FDA) for treatment [1,2,3,4]
The aim of this paper is to report growth response among different diagnostic categories and to identify factors associated with greater growth response over the first 2 years in children with GHD undergoing treatment with GH
Summary
Treatment with exogenous growth hormone (GH) has become a well-accepted therapeutic option for children with growth failure. Considerable variability in response to this treatment has been reported across and within different diagnostic categories [9,10,11]. Such variability makes decisions about whether to treat with GH, when to begin treatment, and what dosing to use more difficult [12]. Variables associated with better responses to GH treatment in patients with ISS include first-year growth response, younger age at start of treatment, the difference in height at the start of treatment from target height SD score (HSDS), and GH dose [13,14]. Additional factors may include underlying genetic conditions, presence of concomitant illness, and compliance with treatment [15]
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