LONG TERM TREATMENT OF BURNED CHILDREN W/ RECOMBINANT HUMAN GROWTH HORMONE

使用重组人生长激素(RhGH)治疗严重烫伤儿童的多中心试验的初步数据表明，这种治疗方法可以加速伤口愈合和改善蛋白质代谢。在主要生理应激源后改善蛋白质动力学的这些明显效果代表了在对抗高分解代谢相关的发病率和死亡率方面的进展的资本收益。目前，还没有关于这类患者长期服用重组人生长激素的初步数据。然而，遭受40%总体表面积烧伤的儿童的长期后遗症包括烧伤后3年内生长速度减慢并伴有永久性发育迟缓。这是一项继续目前的重组人生长激素研究的门诊阶段的建议。这项研究包括急性住院阶段和康复-门诊阶段(本提案)。四家施林纳儿童医院-烧伤研究所将参与其中。我们假设，烧伤后一年内使用重组人生长激素治疗将促进线性生长、骨密度和肌肉。使用最先进的微创技术，我们将在一项双盲研究中评估每年30名烧伤后患者(所有中心的总登记人数)，以确定长期服用重组人生长激素的总体影响。具体地说，我们的目标是确定任何生化或生理证据，证明烧伤导致的分解代谢状态在最初出院后持续存在。这将是第一个关于生长分布和骨密度的大型(多中心)研究，并将作为未来研究烧伤后人群中其他治疗方法的参考。最后，在烧伤后人群中应用rhGH在加速生长速度、全身蛋白质合成、骨骼肌重和恢复正常骨密度方面的长期疗效将被确立。

Preliminary data from a multicenter trial using recombinant human growth hormone (rhGH) in the treatment of children with severe thermal injury suggests that accelerated wound healing and improved protein metabolism occurs with this treatment. These distinct effects of improving protein kinetics after a major physiologic stressor represent a capital gain in the progress against hypercatabolism-associated morbidity and mortality. At present, there are no preliminary data on the long-term administration of rhGH in this patient population. However, the long term sequelae of children suffering a >40% total body surface area burn include a reduction in growth velocity up to 3 years post-burn with permanent stunting. This is a proposal to proceed with the outpatient phase of the current rhGH study. The study consists of an acute-inpatient phase and rehabilitation-outpatient phase(this proposal). Four Shriners Hospitals for Children - Burn Institutes will participate. We hypothesize that treatment with rhGH over a one-year period post-burn will enhance linear growth, bone density and muscle. Using minimally invasive state-of-the-art techniques, we will evaluate 30 (total enrollment from all centers) post-burn patients per year in a double-blind study to determine the overall impact of long-term rhGH administration. Specifically, we aim to identify any biochemical or physiological evidence for persistent after initial hospital discharge of the catabolic state caused by burn injury. This will be the first, large (multi-center) study on growth distribution and bone density and will serve as future reference for studies addressing other treatment approaches in the post-burn population. Finally, the long term efficacy of rhGH administration in the post-burn population with respect to acceleration of growth velocity, whole body protein synthesis, skeletal muscle mass and restoration of normal bone density will be established.