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AbstractObjective:To explore the value of amino-terminal propeptide of C-type natriuretic peptide (NTproCNP) in evaluating the effectiveness of therapy with recombinant human growth hormone (rhGH) in patients with idiopathic short stature (ISS) and isolated growth hormone deficiency (IGHD).Methods:Forty-eight prepubertal children (IGHD=25, ISS=23) treated for at least 1 year with rhGH were included. Insulin growth factor-1 (IGF-I) and NTproCNP serum levels were measured before starting treatment and again 6 months later. Twelve months after starting treatment, all patients were assessed and annual growth velocity (GV), height standard deviation score (HTSDS), and gain HTSDS (ΔHTSDS) were recorded.Results:In the GHD group, positive relationships between GV and change of IGF-ISDS(ΔIGF-ISDS), GV and change of NTproCNP concentrations (ΔNTproCNP) were found. GH peak value was also positively associated with IGF-ISDSand NTproCNP before therapy and ΔIGF-ISDSand ΔNTproCNP were positively associated. In the ISS group, GV was associated with only ΔNTproCNP.Conclusions:NTproCNP is a novel biomarker of growth as levels increase during growth-promoting treatment. Furthermore, IGF-I is also valuable in evaluating the efficacy of rhGH therapy in short stature patients.