生长激素治疗特纳综合征。

4区 医学 Q2 Medicine
Christopher Blunden, Nat Nasomyont, Philippe Backeljauw
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引用次数: 1

摘要

生长衰竭在特纳综合征(TS)患者中几乎是普遍的。它是由于位于X染色体短臂上的矮小同形盒基因(SHOX)的单倍不足造成的。如果不进行治疗,预计患有TS的个体比未受影响的成年女性平均矮20厘米。患者认为身材矮小是他们最大的负担之一,可能对心理社会健康、青春期发育时间和完成各种日常生活活动的能力产生不利影响。常规使用重组人生长激素(rhGH)治疗可增加身高结果。临床证据强烈支持这种治疗的有效性和安全性。在本文中,我们回顾了rhGH治疗TS的基本原理,影响治疗反应的因素,安全性和监测方面的考虑,以及rhGH在未来TS治疗中使用方式的潜在变化。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Growth Hormone Therapy for Turner Syndrome.

Growth failure is nearly universal in individuals with Turner syndrome (TS). It is a consequence of haploinsufficiency of the short stature homeobox gene located on the short arm of the X chromosome (SHOX). Without treatment, individuals with TS are expected to be on average 20 cm shorter than unaffected adult females. Short stature is cited by patients as one of their biggest burdens and may have an adverse impact on psychosocial well-being, pubertal timing, and ability to complete a variety of daily living activities. The routine use of recombinant human growth hormone (rhGH) treatment has increased height outcomes. Clinical evidence has strongly supported the efficacy and safety of this treatment. In this article we review the rationale for rhGH treatment in TS, the factors that affect treatment response, safety and monitoring considerations, and potential changes in the way rhGH may be utilized in TS care in the future.

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来源期刊
Pediatric endocrinology reviews : PER
Pediatric endocrinology reviews : PER Medicine-Endocrinology, Diabetes and Metabolism
自引率
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发文量
1
期刊介绍: PEDIATRIC ENDOCRINOLOGY REVIEWS (PER) publishes scholarly review articles in all areas of clinical and experimental Endocrinology, Diabetes, Nutrition and Metabolism. PER is intended for practicing pediatricians, pediatric endocrinologists, pediatric diabetologists, pediatric gastroenterologists, neonatologists, pediatric gynecologists, nutritionists, sport physicians and pediatricians-in-training. PER will also publish topics on specific subjects or as proceedings of scientific meetings in the above fields of interest. All articles, whether invited or direct contributions, are peer-reviewed. PER publishes correspondence, book reviews, a meeting calendar and meeting reports.
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