Woelfle Joachim, Schnabel Dirk, Binder Gerhard
Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander University of Erlangen-Nuremberg, Erlangen; Centre for Chronic Sick Children, Paediatric Endocrinology, Charité, University Medicine Berlin, Berlin; Pediatric Endocrinology, University Children's Hospital, Universiy of Tuebingen, Tuebingen.
Dtsch Arztebl Int. 2024 Feb 9;121(3):96-106. doi: 10.3238/arztebl.m2023.0247.
3% of all children are unusually short, and 3% are unusually tall. New approaches have broadened the range of therapeutic options in treating growth disorders.
This review is based on publications retrieved by a selective review of the literature and on the authors' clinical experience.
Pituitary growth hormone deficiency is treated with recombinant growth hormone. Long-acting preparations of this type became available recently, but their long-term safety and efficacy are still unknown. Vosoritide, a CNP analogue, has also been approved for the treatment of achondroplasia, and severe primary deficiency of insulin-like growth factor 1 (IGF-1) can be treated with recombinant IGF-1. In the treatment of excessively tall stature, new information on the safety of growth-attenuating treatment and an altered perception of above-average height in society have led to a change in management.
There are new options for the treatment of rare causes of short stature, while new information on the safety of treatment strategies for excessive tallness have led to a reconsideration of surgical intervention. There is insufficient evidence on the benefits and risks of supraphysiological GH therapy and of newer treatment options for which there are as yet no robust data on adult height. Therefore, before any treatment is provided, physicians should give patients and their families detailed information and discuss their expectations from treatment and the goals that treatment can be expected to achieve.
3%的儿童身材异常矮小,3%的儿童身材异常高大。新方法拓宽了治疗生长障碍的治疗选择范围。
本综述基于通过对文献的选择性检索获得的出版物以及作者的临床经验。
垂体生长激素缺乏症采用重组生长激素治疗。这种长效制剂最近已上市,但其长期安全性和有效性仍不清楚。CNP类似物沃索瑞肽也已被批准用于治疗软骨发育不全,严重的原发性胰岛素样生长因子1(IGF-1)缺乏症可用重组IGF-1治疗。在治疗身材过高方面,关于生长减缓治疗安全性的新信息以及社会对高于平均身高看法的改变导致了管理方式的改变。
对于罕见的身材矮小原因有了新的治疗选择,而关于身材过高治疗策略安全性的新信息导致了对手术干预的重新考虑。关于超生理剂量生长激素治疗以及尚无成人身高可靠数据的新治疗选择的益处和风险,证据不足。因此,在提供任何治疗之前,医生应向患者及其家属提供详细信息,并讨论他们对治疗的期望以及治疗有望实现的目标。