Baseline Characteristics and Gender Differences in Prepubertal Children Treated with Growth Hormone in Europe, USA, and Japan: 25 Years' KIGS® Experience (1987-2012) and Review

Background: Information about disease-specific and gender-associated differences over longer time of short children treated with recombinant human growth hormone is missing. Methods: We analyzed data at growth hormone (GH) start in prepubertal children diagnosed with idiopathic GH deficiency (IGHD), congenital GHD, acquired GHD, idiopathic short stature (ISS), and born small for gestational age (SGA) enrolled (1987-2012) in the Pfizer International Growth Study (KIGS®) from Europe, USA, and Japan. Results: The demographic characteristics of patients in the three regions were similar. There was a diagnosis-specific pattern for age and height, and a universal pattern showing that girls were younger and smaller at GH start. There was a predominance of males with IGHD (n = 25,703; 70.1%), congenital GHD (n = 2,860; 63.9%), acquired GHD (n = 3,280; 63.9%), ISS (n = 4,327; 71.4%), and SGA (n = 5,848; 58.0%). Male prevalence in the USA population was more pronounced in IGHD, ISS, and SGA, but less so in congenital and acquired GHD. In IGHD (Europe and Japan) and ISS (Europe), there was a trend toward decreasing male prevalence. Conclusions: The male prevalence in prepubertal children treated with GH varies according to geographical region and is not explained by the underlying diagnoses. A global appreciation of gender biases is required for the proper care of short girls.