Carel J C, Chaussain J L, Chatelain P, Savage M O
INSERUM U342, Hôpital Saint Vincent de Paul, Paris.
Diabetes Metab. 1996 Jul;22(4):251-6.
Growth hormone (GH) insensitivity is a pathological state characterized by a disturbance of the physiological relationship between GH secretion, synthesis of insulin-like growth-factor I (IGF-1) and the biological actions of GH. Laron syndrome, the prototype for GH insensitivity, is most often due to GH receptor deficiency. However, this syndrome is heterogeneous in terms of growth characteristics, bio-chemical features and, most importantly, genetic defects. Recent data have indicated that partial GH receptor deficiency could be involved in children with apparently idiopathic short stature. Laron syndrome, because of extreme growth deficiency and a lack of alternative treatment, was the first clinical situation in which recombinant human IGF-1 was used. IGF-1 accelerates growth rate in most patients, induces subtle modifications of the craniofacies and decreases fat mass. However, it is still too early to evaluate the long-term effects of IGF-1 on final height. Tolerance to the drug has been excellent in all reported trials. The major (but rare) side effects are transient intracranial hypertension and hypokalemia. Generalization of data obtained in Laron syndrome to other clinical situations should take account of the profound alterations in IGF-1 pharmacokinetics resulting from a deficiency in IGF-binding proteins.
生长激素（GH）不敏感是一种病理状态，其特征是GH分泌、胰岛素样生长因子I（IGF-1）合成与GH生物学作用之间的生理关系紊乱。拉伦综合征是GH不敏感的典型病例，最常见的原因是GH受体缺乏。然而，该综合征在生长特征、生化特征，最重要的是在基因缺陷方面具有异质性。最近的数据表明，部分GH受体缺乏可能与明显特发性身材矮小的儿童有关。由于极度生长发育迟缓且缺乏替代治疗方法，拉伦综合征是首个使用重组人生长激素IGF-1的临床病例。IGF-1可使大多数患者的生长速度加快，引起颅面的细微改变，并减少脂肪量。然而，评估IGF-1对最终身高的长期影响仍为时过早。在所有已报道的试验中，对该药物的耐受性都很好。主要（但罕见）的副作用是短暂性颅内高压和低钾血症。将拉伦综合征中获得的数据推广到其他临床情况时，应考虑到IGF结合蛋白缺乏导致的IGF-1药代动力学的深刻改变。
