患有异常性矮身或部分生长激素缺乏症的患者是否需要在青春期继续进行生长激素治疗?
Sena Cantas-Orsdemir1, Adem Orsdemir2, Carly S Barron3
1Department of Pediatrics/Division of Endocrinology, Loma Linda University Children's Hospital, Loma Linda, CA, USA.
Journal of pediatric endocrinology & metabolism : JPEM
|December 29, 2025
概括
在青春期之前的生长激素 (GH) 治疗增加了预测的成人身高 (PAH). 然而,持续GH治疗到青春期并没有显著改善最终的成年人身高,并产生了相当大的成本,这表明需要仔细考虑持续治疗.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 增长激素治疗疗法 增长激素治疗
- 异常发病的矮身症患者.
背景情况:
- 增长激素 (GH) 疗法是建立在增加成年人身高的孩子与异常矮身 (ISS) 和部分GH缺乏症.
- 持续GH疗法的疗效和益处在青春期和青春期内仍然不太清楚.
- 评估青春期开始对预测成年人身高 (PAH) 的影响对于治疗决策至关重要.
研究的目的:
- 评估青春期持续GH治疗是否与青春期开始预测的成人身高 (PAH) 相比改善了成年人身高.
- 为了确定影响青春期GH治疗期间增高的因素.
- 分析长期GH治疗的成本效益.
主要方法:
- 对ISS和部分GH缺乏症患者的多中心回顾性图表审查.
- 在青春期开始之前或在青春期开始时开始GH疗法,并继续直到身高速度下降 (<2.5厘米/年).
- 在各种治疗点收集骨年龄,PAH,高度标准偏差得分 (SDS) 和胰岛素类生长因子1 (IGF-1) SDS.
主要成果:
- 在青春期之前的GH疗法在男孩和女孩中显著增加了PAH.
- 持续GH治疗到青春期没有产生显著的平均身高比PAH在青春期开始 (男孩: -0.62厘米,女孩: 1.82厘米) 的增加.
- 诸如骨年龄,BMI SDS,父母中等身高和青春期治疗的持续时间等因素影响了身高增长,而峰值GH和性别没有预测结果. 在最后一年的GH治疗中,平均成本为122,000美元.
结论:
- 在青春期之前开始的GH疗法有效地增加了预测的成人身高 (PAH).
- 通过青春期继续GH治疗并不总是提供额外的显著的身高益处,并且与高成本有关.
- 临床医生应该仔细考虑潜在的好处与实质性成本,在决定是否继续GH治疗时,一旦达到足够的PAH.
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