ObjectiveThe International Cooperative Growth Study, NutropinAq® European Registry (iNCGS) (NCT00455728) monitored long-term safety and effectiveness of recombinant human growth hormone (rhGH; NutropinAq® [somatropin]) in paediatric growth disorders.MethodsOpen-label, non-interventional, post-marketing surveillance study recruiting children with growth disorders. Endpoints included gain in height standard deviation score (SDS), adult height, and occurrence of adverse events (AEs).Results2792 patients were enrolled. 2082 patients (74.6%) had growth hormone deficiency (GHD), which was isolated idiopathic in 1825 patients (87.7%). Non-GHD diagnoses included Turner syndrome (TS) (n=199), chronic renal insufficiency (CRI) (n=10), other non-GHD (n=498), and missing data for three participants. Improvements from baseline height SDS occurred at all time points to Month 132, and in all subgroups by disease aetiology. At Month 12, mean (95% CI) change in height SDS by aetiology was: idiopathic GHD 0.63 (0.61;0.66), organic GHD 0.71 (0.62;0.80), TS 0.59 (0.53; 0.65), CRI 0.54 (-0.49;1.56), and other non-GHD 0.64 (0.59;0.69). Mean height ( ± SD) at the last visit among the 235 patients with adult or near-adult height recorded was 154.0 cm ( ± 8.0) for girls and 166.7 cm ( ± 8.0) for boys. The most frequent biological and clinical non-serious drug-related AEs were increased insulin-like growth factor concentrations (314 events) and injection site haematoma (99 events). Serious AEs related to rhGH according to investigators were reported (n=30); the most frequent were scoliosis (4 events), epiphysiolysis (3 events), and strabismus (2 events).ConclusionsThere was an improvement in mean height SDS in all aetiology subgroups after rhGH treatment. No new safety concerns were identified.
These results are similar to other Spanish and international published studies, and are representative of the current practice in Spain. Despite treatment being started at a late age, adequate growth is observed in the short term and in the final height. Up to a 24% of patients show a poor response in the first year.
Health related quality of life (HRQoL) is a relevant result when assessing the course of different pathologies and the efficacy of their treatments. HRQoL has been studied previously on adults born small for gestational age (SGA), both in the general population and in patients who had received recombinant human growth hormone (rhGH) treatment, with disparate results. Our study included 50 adults who had received rhGH treatment for the SGA indication in 4 Spanish hospitals. Data have been gathered retrospectively from their clinical records, current weight and height were measured, and patients have been asked to fill out SF-36 and QoLAGHDA quality of life forms, and the Graffar test to evaluate their socio-economical status. Patient’s adult height was – 1.2 ± 0.9 SD, lower than their target height of 1 ± 0.8 SD, but gaining 1.7 ± 1 SD from the beginning of the treatment. SF-36 test results showed lower scoring on Mental Health domains than on those related to Physical Health. No correlation was found between HRQoL results and final height, rhGH treatment duration or puberty. Correlation was indeed found between QoLAGHDA and several domains of SF-36, but QoLAGHDA detected fewer patients with low HRQoL than SF-36. Thus, it is concluded that SGA patient’s follow-up should include a HRQoL, neuro-cognitive and psychiatric assessment in their transition to adult age. Adult SGA patients without catch up growth have impaired HRQoL, especially in mental health domains.
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