Background: Gender may affect growth hormone (GH) treatment outcome. This study assessed gender-related differences in change from baseline height standard deviation scores (ΔHSDS) after 2 years’ GH treatment. Methods: Data from two observational databases were analyzed – the NordiNet® International Outcome Study (NordiNet® IOS) and the American Norditropin Studies: Web Enabled Research Program (ANSWER Program®). Of all the evaluated patients (n = 5,880; age 0 to <18 years), 4,471 were diagnosed with GH deficiency (GHD), 422 with multiple pituitary hormone deficiency, and 987 were born small for gestational age (SGA). Data were analyzed by indication, gender and pubertal status (total population/prepubertal). Results: In the total population, after correcting for dose, mean baseline age and HSDS, ΔHSDS was significantly greater in boys than in girls born SGA (p = 0.0261). In the prepubertal cohort, ΔHSDS was significantly greater for boys versus girls with GHD (p = 0.0004) and SGA (p = 0.0019). No between-gender difference in ΔIGF-I SDS was found. Conclusions: A significant gender difference was found in the 2-year response to GH treatment in the total population of SGA children as well as in the prepubertal cohorts of SGA and GHD children.

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