Effectiveness and Overall Safety of NutropinAq® for Growth Hormone Deficiency and Other Paediatric Growth Hormone Disorders: Completion of the International Cooperative Growth Study, NutropinAq® European Registry (iNCGS).

Regis Coutant,Cristina Patricia Dumitrescu,Dirk Schnabel,Caroline Sert,Jordi Bosch Muñoz,Mehul Dattani,Valerie Perrot

doi:10.3389/fendo.2021.676083

Abstract

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.

Highlights

Growth hormone deficiency (GHD) is characterised by low growth velocity, sometimes after a period of normal growth, and short stature relative to the child’s chronological age, gender and pubertal stage [1]
GH replacement therapy has previously demonstrated efficacy in children treated for GHD and other conditions associated with short stature such as Turner syndrome (TS), chronic renal insufficiency (CRI), and children born small for gestational age [9,10,11,12], with patients with GHD reaching nearadult heights of -0.4 standard deviation (SD) in one study [9]
After treatment with Recombinant human growth hormone (rhGH), height SD score (SDS) increased from baseline at each assessment (Figure 2; Registry population)

Summary

Introduction

Growth hormone deficiency (GHD) is characterised by low growth velocity, sometimes after a period of normal growth, and short stature relative to the child’s chronological age, gender and pubertal stage [1]. GH replacement therapy has previously demonstrated efficacy in children treated for GHD and other conditions associated with short stature such as Turner syndrome (TS), chronic renal insufficiency (CRI), and children born small for gestational age [9,10,11,12], with patients with GHD reaching nearadult heights of -0.4 standard deviation (SD) in one study [9]. A number of registries have assessed the safety and efficacy/effectiveness of the various GH therapies available, in various geographies and for the conditions outlined above [13,14,15,16,17]

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Conclusion