重组人生长激素治疗儿童特发性矮小症远期疗效分析

Long-Term Efficacy of Recombinant Human Growth Hormone in the Treatment of Children with Idiopathic Short Stature

目的:评价重组人生长激素(rh GH)治疗儿童特发性矮小症(ISS)的远期疗效及安全性。方法:连续收集2006年8月至2010年12月在我院就诊的ISS患儿。将愿意接受rh GH治疗的80例患儿纳入治疗组,给予每晚睡前皮下注射rh GH 0.15~0.18 IU/(kg·d),疗程为6个月;另从拒绝接受rh GH治疗的患儿中选取80例年龄、性别及生长发育情况与治疗组患儿相仿的患儿设为对照组,保持自然生长并定期随访。治疗组患儿治疗结束后再随访5年观察治疗效果和不良反应情况。结果:治疗组72例、对照组76例完成5年随访,随访完成率为92.50%(148/160)。随访结束时,治疗组患儿身高从治疗前的(119.27±15.81)cm提高到(151.84±11.53)cm,生长速率从治疗前的每年(3.58±0.57)cm提高到每年(7.45±1.72)cm,且均高于对照组(P均<0.05)。治疗组不良反应/事件发生率为9.72%,予对症处理后不良反应均可消失。结论:rh GH治疗ISS的远期疗效显著,能提高患儿的身高和生长速率,不良反应/事件的发生率较低且可控。

Objective： To evaluate the long-term efficacy and safety of recombinant human growth hormone （rhGH） in the treatment of children with idiopathic short stature （ISS）. Methods： Children with ISS admitted into our hospital from Aug. 2006 to Dec. 2010 were continuously collected. Eighty children who were willing to receive rhGH treatment were enrolled in treatment group, and rhGH 0. 15 0. 18 IU/（ kg·d） was subcutaneously injected before bedtime for a period of 6 months. Another 80 cases with similar age, gender, and growth status were extracted from children who refused to receive treatment as control group, and the control group was maintained natural growth and regular follow-up. After treatment, the treatment group was followed up for 5 years to observe the treatment effects and adverse reactions. Resultys： Seventy-two cases in the treatment group and 76 in the control group completed 5 years of follow-up, with the follow-up completion rate of 92. 50% （148/160）. At the end of follow-up, the height of children with ISS in rhGH treatment group increased from （119.27_＋15.81） cm before treatment to （151.84±11.53） cm, the growth velocity （GV） increased from （3.58±0. 57） cm before treatment to （7.45±1.72 ） cm, and all the data in treatment group were higher than those in control group （P 〈 0. 05 ）. The incidence of adverse reactions/events of treatment group was 9.72%. After the symptomatic treatment, the adverse reactions all disappeared. Conclusion： The long-term efficacy of rhGH in the treatment of ISS is significant, the height and growth rate of children with ISS increase significantly, and the incidence of adverse reactions/events decrease is low and controllable.