重组人生长激素对38例特纳综合征的疗效观察

摘要

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0.5a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±]63.91) 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 discuss the effectiveness and safety of recombinant human growth hormone(rhGH) replacement treatment in growth hormone deficiency ( GHD) children with craniopharyngioma after surgical treatment. Methods This study retrospectively reviewed the records of 12 outpatients with secondary GHD who were treated with rhGH at Department of Neuroendocrinology, Beijing Sanbo Brain Hospital from Apr. 2008 to Apr. 2011. All children (aged 7-15 years) were pathologically diagnosed as craniopharyngioma after surgery, and their follow - up data were included in this analysis. All of the 12 children were given rhGH via subcutaneous injection daily in every evening in original dose of 0.1 IU · kg -1 ,5 days per week,and the course of treatment ranged from 3 to 36 months. Biochemical tests,including liver function,kidney function and plasma hormones concentrations, were arranged regularly. Data of height,weight,growth velocity,standard deviation scoring (SDS), serum insulin - like growth factor 1 (IGF - 1), bone age were recorded and compared before and after the rhGH treatment. Results After one year rhGH replacement therapy,the growth velocities of 12 patients were significantly increased from (2. 2 ± 1. 3) cm · a -1to (6.63 ±4.97) cm · a-1 (P<0.01),SDS had changed from-3.3 ±2.3 to-3.2 ± 2.8(P<0.01) ,and IGF - 1 concentrations were increased from (38 ±64) μg · L -1 to (173 ± 167) μg · L-1 (reaching the normal range)(P <0.01). During the treatment,biochemical tests were in normal range, and there was no speed - up development in the bone age. No tumor recurrence was found by the time of following -up. Conclusions The low dose GH replacement therapy is effective and financially economical in secondary GHD children with craniopharyngioma after surgical treatment. Evaluating and monitoring the states of patients' condition before and during rhGH therapy is necessary to ensure the safety of treatment.