Identification of factors associated with good response to growth hormone therapy in children with short stature: results from the ANSWER Program？

GH-na？ve children with GH deficiency, multiple pituitary hormone deficiency, idiopathic short stature, Turner syndrome, or a history of small for gestational age were eligible (N = 1,002). Using a longitudinal statistical approach, predictive factors were identified in patients with GHD for change from baseline in height standard deviation score (ΔHSDS) following 2 years of treatment.Gradual increases in ΔHSDS over time were observed for all diagnostic categories. Significant predictive factors of ΔHSDS, ranked by significance were: height velocity (HV) at 4 months > baseline age > baseline HSDS > baseline body mass index (BMI) SDS > baseline insulin-like growth factor I (IGF-I) SDS; gender was not significant. HV at 4 months and baseline BMI SDS were positively correlated, whereas baseline age, HSDS, and IGF-I SDS were negatively correlated with ΔHSDS.These results may help guide GH therapy based on pretreatment characteristics and early growth response.Treatment with exogenous growth hormone (GH) has become a well-accepted therapeutic option for children with growth failure. Since the availability of recombinant human GH (rhGH) in 1985, a wide range of conditions associated with decreased growth, including GH deficiency (GHD), Turner syndrome (TS), Noonan syndrome (NS), children born small for gestational age (SGA), Prader-Willi syndrome (PWS), idiopathic short stature (ISS), and SHOX (short stature homeobox) gene haploinsufficiency have been approved by the United States Food and Drug Administration (FDA) for treatment [1-4].Treatment with GH has been demonstrated to increase both short-term growth and adult height in pediatric patients with a variety of different growth disorders [5-8]. However, considerable variability in response to this treatment has been reported across and within different diagnostic categories [9-11]. Such variability makes decisions about whether to treat with GH, when to begin treatment, and what dosing to use more difficult [12].Reports