Abstract
ObjectiveTo investigate the effect of age at growth hormone (GH) treatment start on near adult height (NAH) in children with isolated GH deficiency (GHD).DesignNordiNet® International Outcome Study (IOS) (Nbib960128), a non-interventional, multicentre study, evaluates the long-term effectiveness and safety of Norditropin® (somatropin) (Novo Nordisk A/S) in the real-life clinical setting.MethodsPatients (n = 172) treated to NAH (height at ≥18 years, or height velocity <2 cm/year at ≥16 (boys) or ≥15 (girls) years) were grouped by age (years) at treatment start (early (girls, <8; boys, <9), intermediate (girls, 8–10; boys, 9–11) or late (girls, >10; boys, >11)) and GHD severity (<3 ng/mL or 3 to ≤10 ng/mL). Multiple regression analysis was used to evaluate the effect of age at treatment start (as a categorical and continuous variable) on NAH standard deviation score (SDS).ResultsAge at treatment start had a marked effect on NAH SDS; NAH SDS achieved by patients starting treatment early (n = 40 (boys, 70.0%); least squares mean (standard error) −0.76 (0.14)) exceeded that achieved by those starting later (intermediate, n = 42 (boys, 57.1%); −1.14 (0.15); late, n = 90 (boys, 68.9%); −1.21 (0.10)). Multiple regression analysis showed a significant association between NAH SDS and age at treatment start (P < 0.0242), baseline height SDS (HSDS) (P < 0.0001), target HSDS (P < 0.0001), and GHD severity (P = 0.0012). Most (78.5%) patients achieved a normal NAH irrespective of age at treatment start.ConclusionsEarly initiation of GH treatment in children with isolated GHD improves their chance of achieving their genetic height potential.
Highlights
Bone age was slightly retarded compared with chronological age at treatment start in all three age groups; information on bone age was only available in 48 patients (Table 1)
The results of the present analysis show that in children with isolated GH deficiency (GHD), younger age at treatment start was associated with improved near adult height (NAH) standard deviation score (SDS) and greater ∆height SDS (HSDS) compared with older age at treatment start
Irrespective of age at treatment start, over three-quarters of all patients achieved a NAH SDS within the normal range, albeit at the lower end of the range for those who were older at treatment start
Summary
Treatment with growth hormone (GH) is approved for children with GH deficiency (GHD) to normalise height during childhood and enable the achievement of an adult height within the normal range of the general population (1). Once a diagnosis of GHD has been made it is recommended that treatment with GH is initiated as soon as possible (1). Ross et al reported that among a cohort of older patients (mean (standard deviation (s.d.)) age at GH treatment start 14.0 (2.1) years) with delayed skeletal maturation enrolled in the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program, the majority of GH-treated patients, including those with idiopathic GHD, attained an NAH standard deviation score (SDS) within the normal range (6)
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