Sode-Carlsen Rasmus, Farholt Stense, Rabben Kai Fr, Bollerslev Jens, Schreiner Thomas, Jurik Anne Grethe, Frystyk Jan, Christiansen Jens Sandahl, Höybye Charlotte
Centre for Rare Diseases, Department of Paediatrics, Aarhus University Hospital Skejby, DK-8200 Aarhus N, Denmark.
Growth Horm IGF Res. 2011 Aug;21(4):185-90. doi: 10.1016/j.ghir.2011.05.002. Epub 2011 Jun 12.
Prader-Willi syndrome (PWS) shares similarities with the growth hormone (GH) deficiency syndrome in regards to reduced lean body mass and increased fat mass and several short-term trials with GH treatment have demonstrated beneficial effects on body composition. The aim of the present study was to evaluate the effects and safety of two years of GH therapy in adults with PWS.
Forty-three adults (24 women) with genetically verified PWS were included. Blood samples, body composition as measured by computed tomography (CT) and dual-energy x-ray absorptiometry (DXA) were performed at baseline and during two years of continued GH treatment.
Thirty-nine patients completed treatment for two years. The GH dosage averaged 0.61 mg/day (range 0.2-1.6). Based upon CT, body composition improved at two years; thigh muscle volume increased 6.7 mL (3.7 to 9.7; P<0.001) whereas abdominal subcutaneous fat volume decreased by 53.3 mL (13.8 to 92.9; P=0.01). By DXA, lean body mass improved 2.8 kg (1.9 to 3.6; P<0.001), whereas fat mass decreased by 3.0 kg (1.1 to 4.8; P=0.003). Lung function as evaluated by peak expiratory flow increased 12% (p<0.001) - indicating improved muscle function. Adverse effects were few. Fifteen out of 39 patients had diabetes (DM; n=4) or impaired glucose tolerance (IGT; n=11) prior to GH treatment. Among the 11 patients with IGT, three reverted to normal glucose tolerance, while three progressed to overt DM at two years of GH treatment.
The known beneficial effects of GH treatment upon body composition in PWS are maintained during two years continuous treatment. With appropriate control, GH is a safe treatment option in adults with PWS.
普拉德-威利综合征（PWS）在瘦体重降低和脂肪量增加方面与生长激素（GH）缺乏综合征有相似之处，多项GH治疗的短期试验已证明其对身体成分有有益影响。本研究的目的是评估GH治疗两年对成年PWS患者的疗效和安全性。
纳入43例经基因验证的成年PWS患者（24例女性）。在基线期和持续GH治疗的两年期间进行血样采集，并通过计算机断层扫描（CT）和双能X线吸收法（DXA）测量身体成分。
39例患者完成了两年的治疗。GH剂量平均为0.61mg/天（范围0.2 - 1.6）。基于CT，两年时身体成分得到改善；大腿肌肉体积增加6.7mL（3.7至9.7；P<0.001），而腹部皮下脂肪体积减少53.3mL（13.8至92.9；P = 0.01）。通过DXA测量，瘦体重增加2.8kg（1.9至3.6；P<0.001），而脂肪量减少3.0kg（1.1至4.8；P = 0.003）。通过呼气峰值流速评估的肺功能增加了12%（p<0.001），表明肌肉功能得到改善。不良反应较少。39例患者中有15例在GH治疗前患有糖尿病（DM；n = 4）或糖耐量受损（IGT；n = 11）。在11例IGT患者中，3例恢复为正常糖耐量，而3例在GH治疗两年时进展为显性DM。
在两年的持续治疗中，GH治疗对PWS患者身体成分的已知有益影响得以维持。通过适当控制，GH是成年PWS患者的一种安全治疗选择。
