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一项关于印度身材矮小患者生长激素治疗反应的观察性研究,特别关注生化参数和骨生物标志物。

An Observational Study on Response to Growth Hormone Therapy in Indian Patients of Short Stature with Special Emphasis on Biochemical Parameters and Bone Biomarkers.

作者信息

Roy Ritam, Hazra Avijit, Ghosh Sujoy

机构信息

Department of Pharmacology, IPGME&R, Kolkata, West Bengal, India.

Dean, IPGME&R, Kolkata, West Bengal, India.

出版信息

Indian J Endocrinol Metab. 2023 May-Jun;27(3):260-269. doi: 10.4103/ijem.ijem_303_22. Epub 2023 Jun 26.

DOI:10.4103/ijem.ijem_303_22
PMID:37583404
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10424109/
Abstract

INTRODUCTION

There is a lack of Indian data on short stature treatment using recombinant human growth hormone (rhGH). We explored the effects of such treatment in eastern Indian patients, with emphasis on biochemical parameters and bone biomarkers in addition to basic anthropometry.

METHODS

Our descriptive study covered 50 short stature patients of varied aetiology attending endocrine outpatient department (OPD) of a tertiary care teaching hospital. Patients were followed up for 1 year after the index visit, and prospective data were reconciled with past medical records. A dose of rhGH used was 0.18-0.375 mg/kg as standard, starting dose mostly being 0.2 mg/kg. Dosing was adjusted if the physician judged the clinical outcome to be less favourable than expected. Anthropometric parameters (height, weight, body mass index (BMI) and skeletal age) were recorded clinically, and various biochemical parameters and bone biomarkers were estimated from blood.

RESULTS

Among 50 subjects, 60% had idiopathic growth hormone (GH) deficiency and 26% had Turner's syndrome. The median age at treatment start was 10 years, and the median treatment duration was 25.5 months. The height increased more in the first year of therapy. In the last 6 months, the height velocity was approximately 0.5 cm/month. Although the weight increased significantly, the increment slowed down in the last 6 months. Both remained less than age- and gender-matched references throughout. The skeletal age was on average 2 years behind chronological age (CA)-being 8.7, 9.6 and 11.3 years, respectively, at therapy start, after one year and at study end. Fasting blood glucose (FBG), total cholesterol and calcium level changes were not statistically significant. Serum cortisol and phosphate showed a modest but statistically significant rise, while thyroid-stimulating hormone (TSH) level declined. Insulin-like growth factor 1 (IGF-1) increase was relatively pronounced. Among bone biomarkers, a decrease in CTx and an increase in vitamin D were significant. Dual-energy X-ray absorptiometry (DEXA) data indicated that bone mineral density was less than that of age-matched controls despite treatment. The therapy was well tolerated.

CONCLUSIONS

rhGH treatment leads to significant improvement in anthropometry in Indian children comparable with Western data. Bone biomarker changes indicate decreased bone resorption and increased bone formation although bone mineral density still lags behind age-matched controls.

摘要

引言

关于使用重组人生长激素(rhGH)治疗身材矮小,印度缺乏相关数据。我们探讨了这种治疗方法对印度东部患者的影响,除了基本人体测量学指标外,还重点关注了生化参数和骨生物标志物。

方法

我们的描述性研究涵盖了一家三级护理教学医院内分泌门诊的50名病因各异的身材矮小患者。患者在首次就诊后随访1年,并将前瞻性数据与过去的病历进行核对。rhGH的标准使用剂量为0.18 - 0.375毫克/千克,起始剂量大多为0.2毫克/千克。如果医生判断临床结果不如预期,则调整剂量。临床记录人体测量参数(身高、体重、体重指数(BMI)和骨龄),并从血液中检测各种生化参数和骨生物标志物。

结果

在50名受试者中,60%患有特发性生长激素(GH)缺乏症,26%患有特纳综合征。治疗开始时的中位年龄为10岁,中位治疗持续时间为25.5个月。治疗的第一年身高增长较多。在最后6个月,身高增长速度约为每月0.5厘米。虽然体重显著增加,但在最后6个月增长速度放缓。两者始终低于年龄和性别匹配的参考值。骨龄平均比实际年龄(CA)落后2年,在治疗开始时、治疗1年后和研究结束时分别为8.7岁、9.6岁和11.3岁。空腹血糖(FBG)、总胆固醇和钙水平变化无统计学意义。血清皮质醇和磷酸盐有适度但有统计学意义的升高,而促甲状腺激素(TSH)水平下降。胰岛素样生长因子1(IGF - 1)升高较为明显。在骨生物标志物中,I型胶原交联C末端肽(CTx)下降和维生素D升高具有显著意义。双能X线吸收法(DEXA)数据表明,尽管接受了治疗,但骨矿物质密度仍低于年龄匹配的对照组。该治疗耐受性良好。

结论

与西方数据相比,rhGH治疗可使印度儿童的人体测量学指标显著改善。骨生物标志物的变化表明骨吸收减少和骨形成增加,尽管骨矿物质密度仍落后于年龄匹配的对照组。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/19f9305ee0ad/IJEM-27-260-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/85ad785060d1/IJEM-27-260-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/a6aa9ecd18e4/IJEM-27-260-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/19f9305ee0ad/IJEM-27-260-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/85ad785060d1/IJEM-27-260-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/a6aa9ecd18e4/IJEM-27-260-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5764/10424109/19f9305ee0ad/IJEM-27-260-g003.jpg

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