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生长激素缺乏症和特发性矮小症的生长激素治疗困境:定义、区分与决策

Dilemmas of growth hormone treatment for GH deficiency and idiopathic short stature: defining, distinguishing, and deciding.

作者信息

Halas Julia G, Grimberg Adda

机构信息

College of Arts and Sciences, University of Pennsylvania, Philadelphia, PA, USA.

Division of Endocrinology and Diabetes, Children's Hospital of Philadelphia, Philadelphia, PA, USA -

出版信息

Minerva Pediatr. 2020 Jun;72(3):206-225. doi: 10.23736/S0026-4946.20.05821-1. Epub 2020 Apr 9.

Abstract

Worrisome growth can be a sign of underlying pathology but usually reflects healthy variation. It is often recognized through short stature, which is defined by arbitrarily separating height, a physical trait on a continuum, into "normal" and "abnormal." In some cases of worrisome growth, recombinant human growth hormone (rhGH) treatment is indicated to hasten growth/increase height. This review addresses the two most frequently treated indications for rhGH, growth hormone deficiency (GHD) and idiopathic short stature (ISS). A review of worrisome growth itself, of the history of GH treatment, of the blurry line between partial GHD and ISS, of the GH stakeholders, and of the outside pressures involved in these cases demonstrates the ambiguous platform upon which treatment decisions are made. The rhGH treatment decision process can be examined further by considering the three most impactful factors on parental height-related medical decision-making: treatment characteristics, child health, and psychosocial function. While it is important to note that treatment for classical GHD is uncontroversial and supported, treatment decisions for partial GHD and ISS are more complicated and require careful evaluation of both patient needs and the supporting evidence. As the rhGH community grows, physicians, parents, and patients are encouraged to engage in a shared decision-making process to navigate the many challenges facing the GH field. Although this review addresses GHD and ISS specifically, the issues discussed are often applicable to pediatrics as a whole.

摘要

令人担忧的生长情况可能是潜在病理状况的迹象,但通常反映的是健康变异。它常常通过身材矮小被识别出来,身材矮小是通过将身高(一种连续的身体特征)人为地划分为“正常”和“异常”来定义的。在一些令人担忧的生长情况案例中,需要使用重组人生长激素(rhGH)治疗来加速生长/增加身高。本综述探讨了rhGH最常治疗的两种适应症,即生长激素缺乏症(GHD)和特发性身材矮小(ISS)。对令人担忧的生长情况本身、生长激素治疗的历史、部分GHD和ISS之间模糊的界限、生长激素相关利益者以及这些案例中涉及的外部压力进行综述,揭示了做出治疗决策所依据的模糊平台。通过考虑对与父母身高相关的医疗决策影响最大的三个因素:治疗特征、儿童健康和心理社会功能,可以进一步审视rhGH治疗决策过程。虽然需要注意的是,经典GHD的治疗是毫无争议且得到支持的,但部分GHD和ISS的治疗决策更为复杂,需要仔细评估患者需求和支持证据。随着rhGH领域的发展,鼓励医生、父母和患者参与共同决策过程,以应对生长激素领域面临的诸多挑战。尽管本综述专门讨论了GHD和ISS,但所讨论的问题通常适用于整个儿科学领域。

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