Tas Emir, Uzun Serife E, Tas Vildan, Mejia-Otero Juan D
Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
College of Osteopathic Medicine, New York Institute of Technology, Glen Head, Long Island, NY, USA.
Case Rep Endocrinol. 2021 Jun 17;2021:7865398. doi: 10.1155/2021/7865398. eCollection 2021.
The standard of treatment for pediatric growth hormone deficiency (GHD) is daily subcutaneous recombinant human growth hormone (rhGH) injections. The efficacy of rhGH treatment given as daily intravenous (IV) boluses is not known. . A female with protein C deficiency, a severe bleeding disorder characterized by thrombosis formation, was diagnosed with GHD at age four years. She has been receiving daily protein C infusion through a permanent port since the newborn period. GHD was treated with daily IV rhGH boluses given through the port following protein C infusion. She has reached a growth rate of 12 cm/year and had no side effects. Surprisingly, serum insulin-like growth factor-1 (IGF1) levels did not rise despite an excellent clinical response.
IV administration may be an alternative route for GHD treatment in eligible patients with permanent vascular access. A rise in serum IGF1 levels may not be needed to achieve the growth-promoting effect of rhGH.
儿童生长激素缺乏症（GHD）的标准治疗方法是每日皮下注射重组人生长激素（rhGH）。目前尚不清楚每日静脉推注rhGH治疗的疗效。一名患有蛋白C缺乏症（一种以血栓形成为特征的严重出血性疾病）的女性在4岁时被诊断为GHD。自新生儿期以来，她一直通过永久性端口接受每日蛋白C输注。在输注蛋白C后，通过端口每日静脉推注rhGH对GHD进行治疗。她的生长速度达到了每年12厘米，且没有副作用。令人惊讶的是，尽管临床反应良好，但血清胰岛素样生长因子-1（IGF1）水平并未升高。
对于有永久性血管通路的合格患者，静脉给药可能是GHD治疗的一种替代途径。实现rhGH的促生长作用可能不需要血清IGF1水平升高。
