评价生长激素治疗的反应,当决定治疗时,根据双亲中身高的关系

A. El Awwa, A. Soliman, Suhair Siddig
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Inappropriately, ascribing a child’s extreme shortness to relatively short parents could thus result in delayed investigations or failure to appreciate the seriousness of a child’s situation. Aim The aim of this study is to evaluate the response to growth hormone (GH) therapy when the decision of treatment was made based on their relation to MPH whether FSS or much shorter than their MPH standard deviation score (SDS). Patients and methods This is a retrospective study; we studied the auxologic data of 21 children with FSS [height SDS (HtSDS) <−2, MPHSDS <−2 SD] and 14 children who were not short [HtSDS >−2, but ≥1 SD shorter than their MPHSDS who were treated with recombinant GH (somatropin/norditropin] 0.03–0.05 mg/kg/day daily for more than 1 year, and the dose was adjusted to keep the insulin-like growth factor-1 (IGF-1) level in the upper quartile of normal for age. 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引用次数: 0

摘要

双亲身高(MPH)可作为儿童成人身高的粗略预测指标。家族性身材矮小(FSS)是指与患者年龄、性别和人口相关的最终成年身高低于第三个百分位数的一种情况。然而,在没有营养、激素、获得性、遗传和医源性原因的情况下,它与父母的身高一致。治疗儿童矮小的基本原理包括增加身高和减轻心理社会残疾,同时保持有利的风险/效益和成本/效益比。因此,管理方案之间的选择可能取决于每个方案满足这些目标的程度。不恰当地将孩子的极度矮小归咎于相对矮小的父母,可能会导致调查延迟或无法认识到孩子情况的严重性。目的本研究的目的是评估在决定治疗时对生长激素(GH)治疗的反应,根据他们与MPH的关系,无论是FSS还是远低于他们的MPH标准偏差评分(SDS)。这是一项回顾性研究;我们研究了21例FSS[身高SDS (HtSDS)−2,但比MPHSDS矮≥1 SD的儿童,每日给予0.03 ~ 0.05 mg/kg/day的重组生长激素(生长激素/去甲异丙肽)治疗1年以上,并调整剂量使胰岛素样生长因子-1 (IGF-1)水平保持在正常年龄的上四分位数。结果FSS组GH治疗平均3.27年后,HtSDS升高0.92 SD, IGF-1 SD升高2.87 SD。HtSDS和MPHSDS之间的差异从- 0.18 SD显著改善到0.74 SD。另一组经生长激素治疗后HtSDS升高0.5 SD, IGFSDS升高3.24 SD。HtSDS和MPHSDS的差异从- 1.26 SD显著改善到- 0.72 SD。然而,HtSDS仍然比MPHSDS低0.5以上。两组患者HtSDS增加与GH治疗持续时间呈正相关(r=0.52, P=0.01),与治疗开始时年龄(r= - 0.34, P=0.01)和治疗前IGF-1水平(r= - 0.37, P=0.04)呈负相关。结论生长激素治疗可改善FSS患儿HtSDS,甚至超过MPHSDS。身高正常但远低于MPHSDS的儿童,其HtSDS通过生长激素治疗得到改善,但未达到其MPHSDS。生长激素治疗开始时的持续时间和年龄是获得良好结果的重要参数。MPHSDS是生长激素治疗决策的重要参数。
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Evaluation of the response to growth hormone therapy when the decision of treatment was done based on the relation to the mid parental height
Introduction Midparental height (MPH) can be used as a crude prediction of the child’s adult height. Familial short stature (FSS) is a condition in which the final adult height achieved is less than the third percentile for the patient’s age, sex, and population. Nevertheless, it is consistent with parental height in the absence of nutritional, hormonal, acquired, genetic, and iatrogenic causes. The rationale for treating childhood short stature includes increasing height and alleviating psychosocial disability while maintaining favorable risk/benefit and cost/benefit ratios. Selection among management options may therefore depend on the degree to which each meets these goals. Inappropriately, ascribing a child’s extreme shortness to relatively short parents could thus result in delayed investigations or failure to appreciate the seriousness of a child’s situation. Aim The aim of this study is to evaluate the response to growth hormone (GH) therapy when the decision of treatment was made based on their relation to MPH whether FSS or much shorter than their MPH standard deviation score (SDS). Patients and methods This is a retrospective study; we studied the auxologic data of 21 children with FSS [height SDS (HtSDS) <−2, MPHSDS <−2 SD] and 14 children who were not short [HtSDS >−2, but ≥1 SD shorter than their MPHSDS who were treated with recombinant GH (somatropin/norditropin] 0.03–0.05 mg/kg/day daily for more than 1 year, and the dose was adjusted to keep the insulin-like growth factor-1 (IGF-1) level in the upper quartile of normal for age. Results In the FSS group, after an average of 3.27 years on GH treatment, the HtSDS increased by 0.92 SD, and IGF-1 SD increased by 2.87 SD. Difference between the HtSDS and MPHSDS improved significantly from −0.18 to 0.74 SD. In the other group, after GH therapy the HtSDS increased by 0.5 and IGFSDS increased by 3.24 SD. Difference between the HtSDS and MPHSDS improved significantly from −1.26 to −0.72 SD. However, the HtSDS was still more than 0.5 or more lower than the MPHSDS. In both groups, the HtSDS gain was positively correlated with the duration of GH therapy (r=0.52, P=0.01) and negatively correlated with the age at the start of treatment (r=−0.34, P=0.01) and the pretreatment IGF-1 level (r=−0.37, P=0.04). Conclusion GH therapy improved HtSDS even to exceed MPHSDS in children with FSS. Children with normal stature while far below MPHSDS had their HtSDS improved with GH therapy while did not reach their MPHSDS. The duration and age at the start of GH therapy are important parameters for the favorable outcome. MPHSDS is an important parameter in GH therapy decision.
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