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儿童生长激素缺乏症患者对重组人生长激素(rhGH)治疗的反应。

Response to Recombinant Human Growth Hormone (rhGH) Therapy in Children with Growth Hormone Deficiency.

机构信息

Department of Paediatric Endocrinology, National Institute of Child Health, Karachi, Pakistan.

NIH-Health Research Institute, Research Centre NICH, Karachi, Pakistan.

出版信息

J Coll Physicians Surg Pak. 2024 Aug;34(8):932-935. doi: 10.29271/jcpsp.2024.08.932.

Abstract

OBJECTIVE

To determine the auxological response to recombinant human growth hormone (rhGH) therapy in children with growth hormone deficiency (GHD) presenting at the National Institute of Child Health, Karachi, Pakistan.

STUDY DESIGN

Observational study. Place and Duration of the Study: Department of Paediatric Endocrinology, National Institute of Child Health, Karachi, Pakistan, from January 2022 to December 2023.

METHODOLOGY

All pre-pubertal children with short stature aged 3-12 years diagnosed with GHD and who were prescribed rhGH therapy were included in the study. Children with any other underlying reason for short stature or any other comorbidity were excluded. Patients' demographics and baseline growth parameters were recorded in a pre-designed proforma. Patients were then followed up every three months till one year. Response to rhGH therapy was evaluated through comparison of growth parameters before and after one year of therapy.

RESULTS

A total of 90 children including 47 (52.2%) males and 43 (47.8%) females with GHD were enrolled. Mean age of these patients was 7.92 ± 2.647 years. A statistically significant change in height (SD), Weight (SD), and BMI (SD) was observed before and after one year of therapy (p <0.001). Response to therapy in terms of height did not differ significantly with respect to gender (p = 0.955) or stimulated growth hormone levels (p = 0.911). However, response to rhGH therapy was significantly better in terms of increase in height, weight, and BMI in patients presenting earlier i.e. at age ≤8 years.

CONCLUSION

Recombinant human growth hormone therapy was effective in children with short stature to achieve desirable growth. Children diagnosed and treated at a younger age (≤8years) achieve better height outcomes as compared to those presenting late.

KEY WORDS

Short stature, Growth hormone deficiency, Recombinant human growth hormone.

摘要

目的

确定在巴基斯坦卡拉奇国家儿童健康研究所就诊的生长激素缺乏症(GHD)儿童接受重组人生长激素(rhGH)治疗的生长反应。

研究设计

观察性研究。研究地点和时间:巴基斯坦卡拉奇国家儿童健康研究所儿科内分泌科,2022 年 1 月至 2023 年 12 月。

方法

所有被诊断为 GHD 的 3-12 岁身材矮小的未成熟儿童,如果开处 rhGH 治疗,都被纳入研究。因其他原因导致身材矮小或存在其他合并症的儿童被排除在外。患者的人口统计学和基线生长参数记录在预先设计的表格中。然后,患者每 3 个月随访一次,持续 1 年。通过比较治疗前和治疗 1 年后的生长参数来评估 rhGH 治疗的反应。

结果

共纳入 90 名 GHD 儿童,其中 47 名(52.2%)为男性,43 名(47.8%)为女性。这些患者的平均年龄为 7.92 ± 2.647 岁。治疗前和治疗 1 年后,身高(SD)、体重(SD)和 BMI(SD)均有统计学显著变化(p <0.001)。治疗后身高的反应在性别(p = 0.955)或刺激生长激素水平(p = 0.911)方面没有显著差异。然而,在更早就诊(即≤8 岁)的患者中,rhGH 治疗在身高、体重和 BMI 增加方面的反应更好。

结论

重组人生长激素治疗对身材矮小的儿童有效,可实现理想的生长。在较年幼时(≤8 岁)诊断和治疗的儿童与就诊较晚的儿童相比,身高改善效果更好。

关键词

身材矮小,生长激素缺乏,重组人生长激素。

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