Tauber Maïthé, Diene Gwenaelle, Molinas Catherine
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, Axe Pédiatrique du CIC 9302/INSERM. Hôpital des Enfants, Toulouse, France, Centre de Physiopathologie de Toulouse-Purpan, Université de Toulouse, CNRS UMR 5282, INSERM UMR 1043, Université Paul Sabatier, Toulouse, France.
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, INSERM UMR 1027-Université, Toulouse III Hôpital Paule de Viguier, Toulouse, France.
Pediatr Endocrinol Rev. 2016 Dec;14(2):138-146. doi: 10.17458/PER.2016.TDM.SequelaeofGH.
More than 15 years after rGH was granted marketing authorization for children with PWS, a review of the sequelae, side effects and safety issues of rGH therapy is timely. The publications on issues concerning respiratory function, glucose metabolism, fat mass, and scoliosis at baseline and with rGH treatment are herein presented. We discuss the impact of rGH side effects, make proposals to prevent or treat them, and emphasise the remaining questions and perspectives. As a whole, the benefit /risk ratio is positive, although questions are raised about the role of GH in premature pubarche and its long-term effects, particularly the potential long-term oncogenic risk. The organisation of care in dedicated or reference centres at the national and European level will facilitate the collection and analysis of data and serve as a paradigm for long-term follow-up.
重组人生长激素(rGH)被批准用于普拉德-威利综合征(PWS)儿童治疗15年多后,对rGH治疗的后遗症、副作用和安全问题进行回顾很有必要。本文介绍了关于基线时以及rGH治疗时呼吸功能、葡萄糖代谢、脂肪量和脊柱侧弯等问题的相关出版物。我们讨论了rGH副作用的影响,提出预防或治疗这些副作用的建议,并强调了尚存的问题和前景。总体而言,尽管有人对生长激素在青春期过早出现中的作用及其长期影响,尤其是潜在的长期致癌风险提出质疑,但获益/风险比是积极的。在国家和欧洲层面的专门或参考中心进行护理组织,将有助于数据的收集和分析,并成为长期随访的范例。