重组人生长激素治疗青春晚期大骨龄特发性矮小女童必要性研究

目的 观察骨龄接近闭合的青春晚期特发性矮小(ISS)女童使用基因重组人生长激素(rhGH)治疗的疗效以及用药的必要性,为rhGH临床应用提供依据。方法 43例青春晚期ISS女性患者,骨龄13~15.5岁,根据患儿及家长意愿分为治疗组和对照组。治疗组28例,每晚睡前皮下注射rhGH0.18~0.20 U/kg,共6个月,对照组15例,仅予心理干预及生活指导。记录所有患儿基础及6月后年龄(CA)、身高(Ht)、骨龄(BA),骨龄与生活年龄比(BA/CA)、身高生长速度(GV)、预测成年身高(PH)。6月后计算各组自身预测身高差(PH6-PH0),并问卷家长及患儿满意度。结果 治疗组治疗后生长速度增快,与治疗前自然生长比较,差异有统计学意义(P<0.01);与对照组比较,差异有统计学意义(P<0.01)。预测成人终身高,两组治疗前后及治疗后两组间比较差异均无统计学意义(P＞0.05)。两组自身预测身高差比较差异有统计学意义(P<0.01);对照组6月观察期后预测成人身高小于观察前。满意度调查:治疗组对疗效、医疗费用可接受,对治疗过程认为“值得”、不后悔;对照组反馈“了解过、努力过、是主动放弃的也不后悔”。结论 骨龄接近闭合的青春晚期ISS女童rhGH治疗有效,但不能明显提高终身高。充分医患沟通后,治疗组和对照组对结果有预期,诊治过程对两组近期均产生积极心理影响。青春晚期ISS是否有必要使用rhGH改善身高建议遵从患方意见。

Research of the necessity of rhGH therapy on very late puberty girls with idiopathic short stature.

Objective To assess the efficacy of recombinant human growth hormone (rhGH) therapy on very late puberty girls whose bone aged 13~15.5 with idiopathic short stature (ISS) and discuss the necessary. Methods A total of 43 girls in very late puberty with ISS were divided into control group (n=15) and trial group (n=28) complying with the decision of the girls′ family.The participators in trial group were injected with rhGH 0.18~0.20 U/kg before sleep every night for 6 months,control group were observed for the same time without treatment.Bone age,growth velocity,prediction of adult height were compared between two groups.Totally 43 parents of very late puberty girls were surveyed by the self-administered questions of their own feelings about their own decision of using rhGH injection every day for a long time or not after 6 months. Results There were significant differences in growth velocity between before and after treatment in trial group (P<0.01).And after treatment with rhGH,there was significant difference of the growth velocity between two groups (P<0.01).After 6 months observation,the prediction of adult height was lower in control group,while the result was on the contrary in trial group.There were significant differences between after and before treatment of the value of the prediction of adult height between two groups (P<0.01).In the questionnaire after treatment,the curative effect and medical expense were acceptable for the parents in trial group.And the parents in control group were also calm with their decision of giving up rhGH treatment in the end. Conclusions The rhGH treatment for very late puberty girls can improve the growth velocity but can′t improve the prediction of adult height obviously.With good doctor-patient communications,the diagnosis and treatment process of these very late puberty girls with ISS leaded to a positive psychologic influence on their families both in trial group and control group after 6 months.It was suggested that weather very late puberty girls with ISS should be treated by rhGH should followed the patients′ opinion just on the basis of adequate understanding on their own condition.