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生长激素缺乏症患者治疗第一年反应的评估。

EVALUATION OF FIRST YEAR RESPONSE TO TREATMENT IN CASES WITH GROWTH HORMONE DEFICIENCY.

作者信息

Kör Y, Keskin M

机构信息

Adana Numune Training and Research Hospital, Pediatrics Clinic, Pediatric Endocrinology Division, Adana, Turkey.

Gaziantep University Faculty of Medicine, Dept. of Pediatric Endocrinology, Gaziantep, Turkey.

出版信息

Acta Endocrinol (Buchar). 2016 Oct-Dec;12(4):443-449. doi: 10.4183/aeb.2016.443.

DOI:10.4183/aeb.2016.443
PMID:31149129
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6535253/
Abstract

BACKGROUND/AIMS: Growth hormone (GH) treatment has severe cost burden on patients, their families, and healthcare systems. Therefore, accuracy of diagnosis should be confirmed; factors affecting the response to treatment should be defined. The present study is performed to evaluate auxiliary diagnostic parameters and factors affecting treatment in growth hormone deficiency (GHD).

METHODS

In this study, 142 patients under the age of 16, with at least one year of treatment, were included. Treatment dose of somatropin was 0.2 mg/kg/week in all cases. Response to treatment was evaluated by measuring annual height and height standard deviation score (SDS) gains every 3 months.

RESULTS

Male to female ratio was 79 to 63, and follow-up duration before the treatment was 0.89±0.38 years. Annual growth rate before the treatment was 2.92±1.02 cm, and age at the treatment initiation was 9.97±3.22 years. Height gain SDS at the end of the first year was significantly higher in cases which were at the prepuberty, had severe short stature, low height SDS-mid parental height SDS (HSDS-MPHSDS), and initiated treatment at earlier ages. Correlations in height gain and height SDS gain at the end of the first year were significant between bone age at treatment baseline, delta SDS factors, L-dopa and clonidine stimulation results (both are p<0.01).

CONCLUSION

Height gain was positively related to body mass index, whereas negatively to bone age at treatment baseline, responses obtained from stimulation tests, and delta SDS values. In the treatment evaluation, the parameters which can affect according to model chosen by the investigator, may differ.

摘要

背景/目的:生长激素(GH)治疗给患者及其家庭以及医疗保健系统带来了沉重的成本负担。因此,应确认诊断的准确性;应明确影响治疗反应的因素。本研究旨在评估生长激素缺乏症(GHD)的辅助诊断参数和影响治疗的因素。

方法

本研究纳入了142例16岁以下且接受至少一年治疗的患者。所有病例中生长激素的治疗剂量均为0.2mg/kg/周。每3个月通过测量年身高和身高标准差得分(SDS)的增加来评估治疗反应。

结果

男女比例为79比63,治疗前的随访时间为0.89±0.38年。治疗前的年生长速率为2.92±1.02cm,开始治疗时的年龄为9.97±3.22岁。在青春期前、患有严重身材矮小、身高SDS-父母身高中位数SDS(HSDS-MPHSDS)较低且开始治疗年龄较早的病例中,第一年结束时的身高增加SDS显著更高。治疗基线时的骨龄、SDS变化因子、左旋多巴和可乐定刺激结果之间,第一年结束时的身高增加和身高SDS增加之间存在显著相关性(均为p<0.01)。

结论

身高增加与体重指数呈正相关,而与治疗基线时的骨龄、刺激试验获得的反应以及SDS变化值呈负相关。在治疗评估中,根据研究者选择的模型可能会影响的参数有所不同。

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