生长激素治疗努南综合征患儿的疗效与安全性:证据综述
The efficacy and safety of growth hormone therapy in children with noonan syndrome: a review of the evidence.
作者信息
Noonan Jacqueline A, Kappelgaard Anne-Marie
机构信息
Kentucky Children's Heart Center, Division of Pediatric Cardiology, University of Kentucky, Lexington, Ky., USA.
出版信息
Horm Res Paediatr. 2015;83(3):157-66. doi: 10.1159/000369012. Epub 2014 Dec 10.
Noonan syndrome is a genetic disorder associated with short stature. We reviewed 15 studies in which growth hormone (GH) therapy was used in children with Noonan syndrome. Data show consistent increases in mean height standard deviation score (SDS), with first-year changes of up to 1.26 SDS. Among studies reporting adult or near-adult height, GH therapy over 5-7 years resulted in adult height SDS from -0.6 to -2.1, with up to 60% of subjects in some studies achieving adult height within 1 SDS of mid-parental height. GH treatment results in an acceleration of bone age, likely reflecting normalization from the retarded bone age common in Noonan syndrome patients at the start of therapy. BMI is not affected by GH treatment, but favorable changes in fat mass and body composition are achievable. Longer-term studies and observational studies suggest a waning of the effect of GH therapy over time, as is seen in other GH-treated conditions, and early initiation of therapy and prepubertal status are important predictors of response. GH treatment does not appear to be associated with adverse cardiac or metabolic effects, and data on malignancy during GH treatment give no cause for concern, although they are limited.
努南综合征是一种与身材矮小相关的遗传性疾病。我们回顾了15项关于生长激素(GH)治疗努南综合征患儿的研究。数据显示平均身高标准差评分(SDS)持续增加,第一年变化高达1.26 SDS。在报告成人或接近成人身高的研究中,5至7年的GH治疗使成人身高SDS从-0.6至-2.1,在一些研究中,高达60%的受试者成年身高在父母身高中位数的1个SDS范围内。GH治疗导致骨龄加速,这可能反映了治疗开始时努南综合征患者常见的延迟骨龄恢复正常。BMI不受GH治疗影响,但可实现脂肪量和身体成分的有利变化。长期研究和观察性研究表明,与其他接受GH治疗的情况一样,随着时间的推移,GH治疗的效果会逐渐减弱,治疗的早期开始和青春期前状态是反应的重要预测因素。GH治疗似乎与不良心脏或代谢效应无关,尽管GH治疗期间恶性肿瘤的数据有限,但并无令人担忧的理由。