重组人生长激素用于囊性纤维化的前瞻性随机治疗。
Prospective randomised treatment with recombinant human growth hormone in cystic fibrosis.
作者信息
Schibler A, von der Heiden R, Birrer P, Mullis P E
机构信息
Paediatric Respiratory Medicine, Department of Paediatrics, University of Bern, CH-3010 Bern, Switzerland.
出版信息
Arch Dis Child. 2003 Dec;88(12):1078-81. doi: 10.1136/adc.88.12.1078.
AIM
To evaluate the efficacy and safety of treatment with recombinant growth hormone (rGH) in patients with cystic fibrosis (CF).
METHODS
Twenty patients with CF (aged 10-23 years) were randomised to age and sex matched treatment and control groups. The treatment group received daily subcutaneous injections of 1 IU/kg/wk rGH for 12 months. Pulmonary function (forced expiratory volume in one second (FEV1) and airway resistance), exercise capacity measured with a bicycle ergometer, body composition (dual energy x ray absorptiometry), and weight were assessed at the beginning of the study and after 6 and 12 months.
RESULTS
rGH treatment did not improve weight and pulmonary function, but lean body mass increased significantly in the treatment group. Exercise capacity increased in the treatment group from 143 (16) W (mean (SD)) to 164 (19) W after 12 months of rGH treatment.
CONCLUSION
Treatment of CF patients with rGH for one year improved the exercise capacity significantly but not pulmonary function. The improved exercise capacity needs confirmation in a larger population before such an expensive treatment is justified.
目的
评估重组生长激素（rGH）治疗囊性纤维化（CF）患者的疗效和安全性。
方法
20例CF患者（年龄10 - 23岁）按年龄和性别匹配随机分为治疗组和对照组。治疗组每日皮下注射1 IU/kg/周的rGH，共12个月。在研究开始时以及6个月和12个月后评估肺功能（一秒用力呼气量（FEV1）和气道阻力）、用自行车测力计测量的运动能力、身体成分（双能X线吸收法）和体重。
结果
rGH治疗未改善体重和肺功能，但治疗组瘦体重显著增加。rGH治疗12个月后，治疗组运动能力从143（16）瓦（均值（标准差））增加到164（19）瓦。
结论
用rGH治疗CF患者一年可显著改善运动能力，但不能改善肺功能。在这种昂贵的治疗合理之前，需要在更大规模人群中证实运动能力的改善情况。
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