GROWTH HORMONE REPLACEMENT THERAPY IN CHILDHOOD ONSET HORMONE DEFICIENCY

本研究是一项为期2年的随机开放标签多中心（多国）研究，评价儿童期发作的生长激素缺乏症（GHD）患者在达到成人身高后接受生长激素治疗2年后的体重组成和骨密度。该假说认为，生长激素是需要在成年人维持正常的身体组成。本研究的目的是评估与停止GH治疗的GHD年轻成人相比，骨骺闭合后接受生长激素治疗两年的患者在体重组成方面是否存在任何显著差异。 此外，本研究将评价继续接受标准儿科剂量治疗的儿童与接受标准成人剂量治疗的儿童的身体组成差异。 接受GH治疗直至达到最终身高的儿童将接受成人剂量（0.3 mg rhGH/天）和儿童剂量（0.4 mg/kg/天）治疗2年，并在治疗前后通过DEXA评估体重组成和骨密度。 此时，2例患者入组研究。 第三名患者招募的研究，并开始对rhGH开发了星形细胞瘤复发。 这与药物无关，因为早期脑部MRI显示可能复发。

This study is a randomized open-label multi-center (multi-national) two year study evaluating body mass composition and bone density in patients with childhood onset growth hormone deficient (GHD) after being treated with growth hormone for two years after attaining adult height. The hypothesis is that growth hormone is needed in adults for maintaining normal body composition. The intent of this study is to evaluate whether patients treated with growth hormone for two years after epiphyseal closure show any significant difference in body mass composition as compared to young adults with GHD in whom GH treatment is discontinued. Further, this study will evaluate differences in body composition in children continuing on the standard pediatric doses as compared to those children treated with standard adult doses. Children treated with GH until attainment of final height will be treated with both adult doses (0.3 mg rhGH per day) and pediatric doses (0.4mg/kg/day) for 2 years and body mass composition and bone density assessed by DEXA before and after treatment. At this time 2 patients are enrolled in the study. A third patient recruited for the study and begun on rhGH developed a recurrence of an astrocytoma. This was not drug related because an earlier MRI of the brain demonstrated probable recurrence.