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目的探讨重组人生长激素(rhGH)治疗特纳综合征(TS)的疗效及影响疗效的因素。方法选取确诊为TS的患儿38例为研究对象,每晚均接受rhGH治疗,剂量0.11～0.21 IU.kg-1.d-1,疗程6～36个月。定期随访,观察生长速率(GV)、胰岛素样生长因子-1(IGF-1)和甲状腺功能、肝功能、糖化血红蛋白等。按照rhGH用药剂量将患儿分为2组,≤0.15 IU.kg-1.d-1组和>0.15 IU.kg-1.d-1组,分析不同剂量与疗效的关系。按照治疗初始年龄将患儿分成≤10岁组及>10岁组,分析治疗年龄与疗效的关系。按照治疗前生长激素(GH)峰值将患儿分成GH≥10μg.L-1组及<10μg.L-1组,观察疗效与治疗前GH峰值的关系。结果 TS患儿用药前及用药0.5 a后GV[(2.47±0.42)cm.a-1vs(9.37±2.68)cm.a-1]比较差异有统计学意义(P=0.001)。IGF-1由用药前(186.73±73.32)μg.L-1增至用药后0.5 a(433.93±163.91)μg.L-1,二者比较差异有统计学意义(P<0.001)。rhGH剂量与GV无显著相关。治疗初始年龄与第1年GV无关。治疗前GH峰值与GV无显著相关。结论 rhGH治疗TS可以明确改善身高。较以往常用剂量更小的剂量也可以达到很好的疗效。用药前患儿GH峰值与疗效关系不大,可以不用常规做GH激发试验。患儿治疗的初始年龄与GV无明显相关性。IGF-1是治疗过程中的重要检测指标。
Objective To investigate the efficacy and safety of recombinant human growth hormone (rhGH) in the treatment of Turner syndrome (TS). Methods Thirty-eight children diagnosed with TS were enrolled in this study. Each night rhGH was given at a dose of 0.11-0.21 IU.kg-1.d-1 for 6 to 36 months. Regular follow-up, growth rate (GV), insulin-like growth factor-1 (IGF-1) and thyroid function, liver function, glycosylated hemoglobin and so on. The patients were divided into two groups according to rhGH dosage, ≤ 0.15 IU.kg-1.d-1 group and> 0.15 IU.kg-1.d-1 group. The relationship between different doses and curative effect was analyzed. According to the initial treatment age, the children were divided into groups of ≤10 years old and> 10 years old. The relationship between treatment age and curative effect was analyzed. The patients were divided into GH≥10μg.L-1 group and <10μg.L-1 group according to the peak value of growth hormone (GH) before treatment, and the relationship between therapeutic effect and pre-treatment GH peak was observed. Results There was significant difference in GV [(2.47 ± 0.42) cm.a-1 vs (9.37 ± 2.68) cm.a-1 before treatment and after 0.5 a) in TS children (P = 0.001). IGF-1 increased from (186.73 ± 73.32) μg.L-1 before treatment to 0.53 (433.93 ± 163.91) μg.L-1 after treatment, the difference was statistically significant (P <0.001). There was no significant correlation between dose of rhGH and GV. The initial treatment age was independent of the first year GV. The peak of GH before treatment was not significantly correlated with GV. Conclusion rhGH treatment of TS can clearly improve the height. More than the usual dose of smaller doses can achieve good results. Before treatment, the relationship between the peak value of GH in children and the curative effect is not significant, GH stimulation test can be done without routine. There was no significant correlation between the initial age of treatment and GV in children. IGF-1 is an important indicator of the course of treatment.