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Growth hormone therapy in children and adolescents: pharmacokinetic/pharmacodynamic considerations and emerging indications.

机译:儿童和青少年的生长激素治疗:药代动力学/药效学方面的考虑和新出现的适应症。

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摘要

Human growth hormone (hGH) has been used for GH deficiency (GHD) for more than 40 years. The availability of hGH produced using recombinant DNA technology beginning in 1985 both eliminated the infectious risk of pituitary-derived hGH and provided a greatly increased supply, allowing for an expanded range of indications. Approved pediatric indications include GHD, idiopathic short stature, children who were small for gestational age with short stature, Turner syndrome, Prader-Willi syndrome, short stature homeobox-containing gene deficiency, Noonan syndrome and chronic renal insufficiency. This review will summarize our current understanding of the pharmacokinetics and pharmacodynamics of hGH, with an emphasis on aspects relevant to recent investigational uses in pediatrics. Promising areas of investigation include cystic fibrosis, juvenile idiopathic arthritis and inflammatory bowel disease. However, given the high cost and potential for rare side effects, controversy remains about the optimal indications, dose and duration of hGH therapy for both the approved and investigational conditions.
机译:人类生长激素(hGH)已被用于治疗GH缺乏症(GHD)40多年了。从1985年开始使用重组DNA技术生产的hGH不仅消除了垂体来源的hGH的感染风险,而且大大增加了其供应,从而扩大了适应症范围。批准的儿科适应症包括GHD,特发性矮小身材,身高矮小,胎龄矮的儿童,特纳综合症,Prader-Willi综合症,含有矮身形同源基因的基因缺乏症,Noonan综合症和慢性肾功能不全。这篇综述将总结我们目前对hGH的药代动力学和药效学的理解,重点是与近期在儿科研究用途相关的方面。有希望的研究领域包括囊性纤维化,青少年特发性关节炎和炎症性肠病。然而,考虑到高成本和潜在的罕见副作用,对于已批准和研究条件下的hGH治疗的最佳适应症,剂量和持续时间仍存在争议。

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