重组 GH 疗法:儿童的预期结果、治疗方式和监测。

Maria Elisa Amodeo, Cesare Morgante, Valentina Pampanini, Annalisa Deodati, Stefano Cianfarani
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摘要

上个世纪,生长激素(GH)治疗经历了从垂体衍生生长激素(可用于治疗特定的生长激素缺乏症(GHD))到重组人生长激素(rhGH)的演变过程。经证实,使用 rhGH 治疗可有效改善 GHD 患者身材矮小的情况,同时还对新陈代谢和骨骼健康产生有益影响。对rhGH治疗的长期安全性进行了深入研究,结果令人欣慰,尤其是对接受孤立性GHD治疗的患者。在GHD以外的其他疾病中使用超生理剂量的rhGH,以及与某些临床症状相关的内在风险因素,都促使我们需要延长对rhGH安全性的监测研究。最后,长效rhGH制剂正在进入市场,其目标是提高rhGH治疗的依从性并减轻患者的治疗负担。在本综述中,我们将讨论rhGH治疗GHD儿科患者的适应症、疗效和安全性。我们还将简要讨论rhGH的新配方。
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Recombinant GH Therapy: Expected Results, Modalities and Monitoring in Children.

The past century witnessed the evolution of growth hormone (GH) treatment from pituitary-derived GH, available for treatment of selected cases of GH deficiency (GHD), to recombinant human GH (rhGH), currently approved for several clinical disorders besides GHD. Treatment with rhGH has a proven efficacy in improving short stature in patients affected by GHD, alongside beneficial effects on metabolic and bone health. Long-term safety of rhGH treatment has been intensively investigated, providing reassuring results, especially in patients treated for isolated GHD. The use of rhGH in supraphysiological doses in conditions other than GHD and the intrinsic risk factors associated with certain clinical conditions prompt the need to prolong surveillance studies on rhGH safety. Finally, long-acting rhGH formulations are being introduced into the market, with the goal of increasing adherence and reducing treatment burden for patients on rhGH treatment. In this review, we will discuss indications, efficacy, and safety profile of treatment with rhGH in pediatric patients affected by GHD. We will also briefly discuss the newer formulation of rhGH.

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