Abstract
BackgroundA primary goal of recombinant human growth hormone therapy (GHT) in children is attaining normal adult height. In this study, children with growth hormone deficiency (GHD) (including isolated idiopathic growth hormone deficiency [IGHD] and multiple pituitary hormone deficiency [MPHD]), idiopathic short stature (ISS), and Turner syndrome (TS) were evaluated for near-adult height (NAH) and percent achieving NAH within the normal range after approximately 4 years of GHT.MethodsData from the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program were analyzed for NAH from age at treatment start (ATS) (i.e., referral age as defined by age at enrollment in the study) to last clinic visit using one of the following two criteria: 1) age ≥18 years, or 2) if male: ≥16 years and height velocity (HV) <2 cm/year; if female: ≥15 years and HV <2 cm/year. All patients had a baseline height standard deviation score (HSDS) ≤ -2, and either GHD (n = 201), ISS (n = 19), or TS (n = 41). The main outcome measures included HSDS and corrected HSDS (HSDS-target HSDS) in response to GH treatment, and correlation of ATS with NAH HSDS.ResultsMean (± SD) chronological and bone ages at baseline were 14.0 ± 2.1 years and 11.7 ± 2.0 years, respectively, and mean GHT duration was 4.0 ± 1.6 years. Mean HSDS (baseline to NAH; GHD: -2.7 to -1.0; ISS: -2.8 to -1.4; TS: -3.0 to -1.8) and mean corrected HSDS (baseline to NAH; GHD: -2.1 to -0.3; ISS: -2.1 to -0.6; TS: -1.8 to -0.6) increased across diagnostic indications. Percentages of patients reaching near-adult HSDS > -2 were GHD: 87.6%; ISS: 78.9%; TS: 65.8%. Significant negative correlations were found between ATS and NAH HSDS when analyzed by sex.ConclusionsDespite a relatively advanced childhood age, the majority of GH-treated patients attained mean near-adult HSDS within the normal range (HSDS > -2). Negative correlations of ATS with near-adult HSDS indicate that an earlier age at treatment start would likely have resulted in greater adult height achieved in both male and female patients.
Highlights
A primary goal of recombinant human growth hormone therapy (GHT) in children is attaining normal adult height
The use of GH is approved by the US Food and Drug Administration for the treatment of growth failure associated with growth hormone deficiency (GHD), either isolated (IGHD) or part of multiple pituitary hormone deficiency (MPHD), and short stature associated with Turner syndrome (TS), Noonan syndrome (NS), children born small for gestational age (SGA), short stature homeobox (SHOX) gene haploinsufficiency, Prader-Willi syndrome (PWS), chronic kidney disease (CKD), or idiopathic short stature (ISS) [1]
Patient demographics and baseline characteristics A total of 261 GH-naïve patients who were diagnosed with GHD (n = 201), ISS (n = 19), or TS (n = 41), and had achieved near-adult height (NAH), were included in the analyses
Summary
A primary goal of recombinant human growth hormone therapy (GHT) in children is attaining normal adult height. Children with growth hormone deficiency (GHD) (including isolated idiopathic growth hormone deficiency [IGHD] and multiple pituitary hormone deficiency [MPHD]), idiopathic short stature (ISS), and Turner syndrome (TS) were evaluated for near-adult height (NAH) and percent achieving NAH within the normal range after approximately 4 years of GHT. Attaining adult height within the normal range is an important treatment goal for GHT in children with short stature or growth failure disorders, especially with respect to the potential benefits in health-related quality of life (HRQoL) and physical and psychosocial wellbeing [5,6]. A multicenter, randomized, doubleblind clinical study of children with short stature who were born SGA without signs of catch-up growth showed that GH treatment to adult height was associated with a significant improvement in HRQoL and the normalization of final height [5]
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