Recombinant Human Growth Hormone Plus Recombinant Human Insulin-Like Growth Factor-1 Coadministration Therapy in Short Children with Low Insulin-Like Growth Factor-1 and Growth Hormone Sufficiency: Results from a Randomized, Multicenter, Open-Label, Parallel-Group, Active Treatment-Controlled Trial

摘要

Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh) GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score <=-2.0 and IGF-1 SD score <=-1.0 for age and sex, and with stimulated GH >= 10 ng/ml for age and sex, were randomized to receive ( all doses in mu g/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and. height SD score were measured. Results: The first-year HV (modified intention-totreat population) was 9.3 +/- 1.7 cm/year (group A), 10.1 +/- 1.3 cm/year (group B), 9.7 +/- 2.5 cm/year (group C) and 11.2 +/- 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, co-administration of rhGH/rhIGF-1 (45/150 mu g/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies. (C) 2015 S. Karger AG, Basel