Tidblad Anders, Sävendahl Lars
Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden; Division of Paediatric Endocrinology, Karolinska University Hospital, Stockholm, Sweden.
Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden; Division of Paediatric Endocrinology, Karolinska University Hospital, Stockholm, Sweden.
Lancet Child Adolesc Health. 2024 Aug;8(8):600-610. doi: 10.1016/S2352-4642(24)00127-5. Epub 2024 Jun 27.
With long standing demand and popularity, growth hormone treatments continue to be a topic of interest for paediatric endocrinologists and general paediatricians due to ongoing issues regarding their long-term effects, the safety of childhood treatment, and the introduction of long-acting growth hormone preparations in the past decade. Moreover, uncertainty regarding how to approach individual patients and their treatment indications remains, particularly concerning tailored treatment goals and objectives; this uncertainty is further complicated by the multitude of approved indications that surpass substitution therapy. The paediatric endocrinologist thus grapples with pertinent questions, such as what defines reasonable treatment goals for each individual given their indications, and when (and how) to initiate the necessary discussions about risks and benefits with patients and their families. The aim of this Review is to offer advanced physiological concepts of growth hormone function, map out approved paediatric indications for treatment along with evidence on their effects and safety, highlight controversies and complexities surrounding childhood growth hormone treatment, and discuss the potential of long-acting growth hormone and future directions in the realm of childhood growth hormone treatment.
由于生长激素治疗长期存在需求且广受欢迎，加之其长期影响、儿童期治疗安全性以及过去十年长效生长激素制剂的引入等持续存在的问题，该治疗仍然是儿科内分泌学家和普通儿科医生感兴趣的话题。此外，对于如何治疗个体患者及其治疗指征仍存在不确定性，尤其是在定制治疗目标方面；众多已批准的适应症超越了替代疗法，这使得这种不确定性更加复杂。因此，儿科内分泌学家面临着一些相关问题，比如根据每个患者的指征，什么才是合理的治疗目标，以及何时（以及如何）与患者及其家属就风险和益处展开必要的讨论。本综述的目的是提供生长激素功能的先进生理学概念，梳理已批准的儿科治疗适应症及其疗效和安全性证据，突出儿童生长激素治疗周围的争议和复杂性，并讨论长效生长激素的潜力以及儿童生长激素治疗领域的未来发展方向。
