title: Comparative evaluation of short-term biomarker response to treatment for growth hormone deficiency in Chinese children with growth hormone deficiency born small for or appropriate for gestational age: a randomized phase IV open-label study.
abstract: OBJECTIVE
  To compare the response between Chinese children with growth hormone deficiency (GHD) born either small for gestational age (SGA) or appropriate for gestational age (AGA) after 4 weeks of recombinant human growth hormone (r-hGH) therapy.
  
  
  METHODS
  This was a phase IV, open-label, multicenter, interventional study (NCT01187550). Prepubertal children with GHD received open-label treatment with daily r-hGH (0.033 mg/kg) for 4 weeks. Serum levels of insulin-like growth factor I (IGF-I) and insulin-like growth factor-binding protein 3 (IGFBP3), and metabolic markers (including fasting glucose, insulin, total cholesterol, and homeostasis model assessment of insulin resistance) were assessed at baseline and after 4 weeks of treatment, and were analyzed according to patient subgroup (SGA or AGA).
  
  
  RESULTS
  A total of 205 children with GHD (mean age 10.4 years; 175 AGA, 30 SGA) were included in the analysis. Mean baseline serum IGF-I and IGFBP3 standard deviation scores (SDS) across the whole patient population were lower than the population norms (mean values: -2.1 SDS for IGF-I and -1.2 SDS for IGFBP3), with no significant differences between the two patient subgroups. After 4 weeks, IGF-I and IGFBP3 levels increased by 1.0 SDS (p < 0.001) and 0.34 SDS (p < 0.001), respectively, but no significant differences were found between the two patient subgroups for growth-related or metabolic markers.
  
  
  CONCLUSIONS
  For children with GHD born SGA, IGF-I and IGFBP3 are short-term biomarkers of responsiveness to treatment with growth hormone, as for children with GHD born AGA.
authors.affiliation: Ruijin Hospital, Jiaotong University School of Medicine, Shanghai, China.
authors.forename: Wenli
authors.initials: W
authors.lastname: Lu
authors.forename: Shuixian
authors.initials: S
authors.lastname: Shen
authors.forename: Xiaoping
authors.initials: X
authors.lastname: Luo
authors.forename: Chunxiu
authors.initials: C
authors.lastname: Gong
authors.forename: Xuefan
authors.initials: X
authors.lastname: Gu
authors.forename: Yun
authors.initials: Y
authors.lastname: Li
authors.forename: Minlian
authors.initials: M
authors.lastname: Du
authors.forename: Runming
authors.initials: R
authors.lastname: Jin
authors.forename: Queena
authors.initials: Q
authors.lastname: Zhou
authors.forename: Wei
authors.initials: W
authors.lastname: Wang
keywords: biomarker response; insulin-like growth factor I; insulin-like growth factor-binding protein 3; recombinant human growth hormone