重组人生长激素长疗程治疗特发性矮小症疗效观察

目的探讨基因重组人生长激素(recombinant human growth hormone,rhGH)对特发性矮小症(idiopathic shortstature,ISS)儿童长疗程治疗的疗效。方法 60例ISS患儿(男性21例,女性39例),每晚皮下注射rhGH 0.15～0.18IU/(kg.d),疗程1～3年。评价治疗前后身高、生长速度、身高标准差计数(SDS)、骨龄、预测成年身高变化及副作用。结果①rhGH治疗1～3年期间,60例患儿生长速率由治疗前的每年(4.51±0.46)cm分别提高到(10.97±2.53)、(8.11±1.54)cm和(7.13±2.07)cm;身高SDS由治疗前(-2.60±0.57)增至(-1.62±0.64)、(-1.29±0.89)及(-0.12±0.45);预测成人身高由治疗前(151.62±8.46)cm分别增加为(157.33±8.50)、(160.72±10.31)cm和(165.81±7.46)cm(P<0.05)。②不同青春期开始治疗的疗效比较,TannerⅠ、Ⅱ、Ⅲ期患儿生长速率相接近,明显高于Ⅳ期。③骨龄增长低于身高年龄增长(P<0.05),而与年龄增长相一致(P>0.05)。结论长疗程rhGH治疗对ISS有明显的促生长效应,增加了预测成年身高;青春期前、青春早中期开始治疗的疗效优于青春晚期;长疗程的rhGH治疗未引起骨龄和青春期提前。

Efficiency of long-term recombinant human growth hormone therapy for children with idiopathic short stature

Objective To assess the efficiency of recombinant human growth hormone(rhGH) for a long-term therapy for children with idiopathic short stature(ISS).Methods Sixty ISS children(21 males and 39 females) were treated with rhGH 0.15 to 0.18 IU/kg daily by subcutaneous injection at periumbilical area or lateral thigh for 1 to 3 years.Changes of height,growth velocity,mean height standard deviation score(SDS),bone age and predicted adult height before and in every 6 month after therapy were measured.Side effects were evaluated.Results With different courses of treatment,the mean growth velocity was increased from(4.51±0.46) cm/year to(10.97±2.53),(8.11±1.54)cm/year and(7.13±2.07)cm/year respectively for those children treated for 1,2 and 3 years.Mean height SDS was increased form(-2.60±0.57) to(-1.62±0.64),(-1.29±0.89) and(-0.12±0.45) respectively.Predicted adult height was increased form(151.62±8.46)cm to(157.33±8.50),(160.72±10.31)cm and(165.81±7.46)cm respectively(P<0.05).The growth velocity of ISS children in Tanner stage Ⅰ,Ⅱ and Ⅲ were similar and significantly better than that of the stage Ⅳ.The acceleration of height age was faster than the bone age(P<0.05),whereas the acceleration of bone age was as fast as chronological age(P>0.05).Conclusion Long-term therapy with rhGH for ISS children is of significant effectiveness in improving growth and increasing prediction adult height.The onset of therapy starting in children in the prepuberty,earlier and middle puberty is more effective than the latter puberty.Long course of rhGH treatment does not advance the puberty and bone age.