GROWTH HORMONE REPLACEMENT THERAPY IN CHILDHOOD ONSET GROWTH HORMONE DEF

国际多中心随机对照试验，研究生长激素替代（hGH）对儿童期生长激素缺乏症（GHD）的青少年和年轻成人患者先前治疗至最终身高的影响。先前从儿童期发病的成人GHD患者中获得的数据表明，尽管接受生长激素治疗直到骨骺闭合，但这些患者的最终身高结果令人满意，但可能没有达到其他发育里程碑。有证据表明，通常要到骨骺闭合后，即达到最终高度后，才能达到成人体型和骨量峰值。因此，仅治疗至最终身高的患者往往瘦体重低于正常水平，骨矿物质含量（BMC）和骨矿物质密度（BMD）低于正常水平。本临床研究将验证骨骺闭合后继续生长激素替代治疗两年将导致瘦体重、BMC和骨密度增加的假设。还将评估成人接受的每日替代剂量（0.017-0.034IU/kg/天），以确定其是否足以覆盖年轻人骨骺闭合后的发育阶段，或者更大的剂量（0.070-0.100 IU/kg/天）是否更合适。将包括三个治疗组；儿童剂量，成人剂量，无治疗。

International multi-center randomized controlled trial to examine the effect of growth hormone replacement (hGH) in adolescent and young adult patients with childhood onset growth hormone deficient (GHD)previously treated to final height. Previous data obtained from adult GHD patients of childhood onset treated with hGH until epiphyseal closure indicate that in spite of satisfactory final height results, other developmental milestone may not have been achieved in these individuals. Evidence suggests that the attainment of adult body shape and of peak bone mass are usually not reached until after epiphyseal closure, i.e. after final height has been reached. Thus, patients only treated to final height often have a lower than normal lean body mass, and a lower than normal bone mineral content (BMC) and bone mineral density (BMD). This clinical study will test the hypothesis that continuing hGH replacement therapy for two years after epiphyseal closure will result in increased lean body mass, BMC, and BMD. The accepted daily replacement dosage for adults (0.017-0.034IU/kg/day) will also be evaluated to see if it is significant enough to cover the developmental phase following epiphyseal closure in young adults, or if a larger dosage closer to that used in children (0.070-0.100 IU/kg/day) is more appropriate. Three treatment arms will be included; pediatric dosage, adult dosage, and no treatment.