重组人生长激素对生长激素缺乏症患儿成年身高的影响

Effect of recombinant human growth hormone on final adult height in children with growth hormone deficiency

目的观察重组人生长激素(rhGH)对生长激素缺乏症(GHD)患儿成年身高(FAH)的影响。方法对11例GHD患儿(男7,女4)应用rhGH治疗,每晚睡前皮下注射,剂量为0．7 IU·kg^(-1)·w^(-1),疗程1．1～7．3年。治疗结束后定期复诊,年生长速度＜1cm,或女性患儿骨龄(BA)≥14岁,男性患儿BA≥16岁时的身高视为近似成年身高或FAH。分析FAH和影响FAH的因素。结果FAH标准差分值(SDS)在男性患儿为(-1．47±0．37),女性患儿为(-1．07±0．60)。85．7%(6/7)男性患儿的FAH达到或超过遗传靶身高(THt)范围,50%(2/4)女性患儿的FAH达到或超过THt范围,男女患儿的FAHSDS与THtSDS比较差异无统计学意义(P＞0．05)。男性患儿的FAHSDS与治疗开始的年龄呈负相关,与治疗开始按年龄的身高SDS(HtSDS_(CA))、青春发育时HtSDS_(CA)和疗程呈正相关；逐步回归分析显示,青春发育时HtSDS_(CA)是影响男性患儿FAHSDS(F=32．58,P=0．002)的独立因素。而女性患儿的FAHSDS与上述因素不相关。孤立性GHD患儿的FAHSDS与由垂体病变引起的多激素缺乏症患儿相比差异无统计学意义(P＞0．05)。结论rhGH能改善GHD患儿的FAH。影响FAH的最主要因素是青春发育时的身高。因此,为达到满意的成年身高,对GHD患儿必须尽早诊断,尽早治疗,以使在青春发育前达到较理想的身高,而对于青春期开始治疗者,必须采用足够的剂量以获得较好FAH。

Objective To evaluate the effect of recombinant human growth hormone （rhHG） on the final adult height （FAH） in the children with growth hormone deficiency （GHD）. Methods This study included ll children （7 boys and 4 girls） with GHD, who were treated with subcutaneous injections of rhGH 0.7 IU· kg^-1 · w^-1 daily before sleep for 1. 1 to 7. 3 years. Regular follow-up observation was taken after treatment being discontinued. FAH was defined as a bone age ≥16 years for boys and ≥14 years for girls and the growth rate less than 1 cm/year. FAH and the factors determining FAH were also evaluated. Results FAH standard deviation score （FAHSDS） were （-1.47±0.37） in boys and （-1.07±0.60） in girls. 85.7% （6/7） of the boys and 50% （2/4） of the girls achieved FAH height which was comparable to or above their genetic target height （THt）. FAHSDS was not significantly different from THt standard deviation score （THtSDS） in boys and girls （ P 〉 0.05 ）. FAHSDS in boys was negatively correlated with the age at the initiation of therapy and was positively correlated with HtSDS at the initiation of GH therapy, HtSDS at initiation of puberty and duration of therapy. Stepwise regression analyse indicated that HtSDS at start of puberty was the variable with the greatest identified influence on FAHSDS in boys （F =32.58, P = 0. 002）. But FAHSDS in girls was not correlated with the above variables. There was no difference in FAHSDS between children with isolated GHD and those with multiple hormone deficiencies （ P 〉 0.05 ）. Conclusion GH improves FAH of children with GHD. Height at the initiation of puberty is the most significant determining factor for the long-term efficacy. Hence, it is important that the diagnosis should be made and treatment be initiated as early as possible to afford children with GHD the opportunity to make up much of their height deficit before puberty. Adequate dosage of GH should be used for the children taking initial treatment at puberty to attain satisfactory FAH.